When you think of Parkinson's disease (PD), what likely comes to mind are the physical symptoms—tremors, stiffness, and slowness of movement. These are the visible signs of a disease that affects nearly one million people in the US alone. But there's another side to Parkinson's that often remains in the shadows, a side that doesn't always get the attention it deserves: the mental and emotional challenges.

If you or a loved one is living with Parkinson's, you may already know that it's not just the physical symptoms that are tough to deal with. The emotional rollercoaster, the anxiety, the creeping feelings of depression, and even cognitive struggles can be just as overwhelming, if not more so. These hidden struggles can make an already challenging journey even more difficult.

Yet, despite how common these mental health issues are for those with Parkinson's, they often go unspoken. It's easy to focus on what you can see—the shaking hands, the slow steps—but what about the things you can't see? The feelings of isolation, the fear of what comes next, the frustration when your mind doesn’t seem as sharp as it used to be?

An Overview of Parkinson’s Diseases

Parkinson's disease is one of the most common movement disorders. It mostly affects your motor function, which includes your ability to control and coordinate movements. This disease occurs when certain nerve cells in a specific part of the brain called the substantia nigra start to break down or die.

These nerve cells are responsible for producing dopamine, a neurotransmitter that plays a critical role in coordinating smooth and controlled muscle movements. As the dopamine levels decrease, the ability to control movement becomes impaired, leading to the characteristic symptoms of PD, which include:

• Tremors
• Slowness of movement
• Muscle rigidity
• Postural instability
• Gait changes
• Decreased facial expression
• Speech and writing changes

But beyond the hallmark motor function symptoms, you may also experience non-motor symptoms like:

• Cognitive decline
• Mood disorders
• Autonomic nervous system dysfunction (constipation, orthostatic hypotension, sexual dysfunction, etc.)
• Fatigue
• Sleep dysfunction
• Loss of sense of smell (anosmia)
• Visual hallucinations

These non-movement symptoms are common and affect the quality of life. In fact, according to the largest-ever clinical study of Parkinson’s by the Parkinson’s Foundation, depression and anxiety are the leading factors that affect the overall health of people living with the condition.

The Mental Health Aspects of Parkinson’s

Depression, anxiety, cognitive changes, psychosis are all extremely common in Parkinson’s disease. Several large studies have revealed the magnitude of these symptoms. More than 50% of all Parkinson’s patients experience mental illness at some point in the disease. Sadly, studies have also shown that physicians often miss these signs, and even when they recognize them, they sometimes don’t treat them.

Parkinson’s and Depression

Many people experience grief or sadness when they get a diagnosis of a chronic condition like Parkinson’s disease. But depression is different. Unlike sadness which is temporary, depression lasts for weeks or months. Depression is a part of Parkinson’s itself, arising from Parkinson’s-related changes in the brain.

You may experience depression at any time in the course of Parkinson’s, even before diagnosis. And the depression symptoms may come and go. An older study found that people with a depression diagnosis were 3.24 times more likely to develop PD than those without. Another study established that depression might be an early sign or a risk factor for PD.

PD affects areas of the brain that produce dopamine, serotonin and norepinephrine – neurotransmitters that regulate mood, appetite, motivation and sleep. Experts believe that an imbalance in these chemicals is what contributes to depression. However, other factors such as negative thinking, social isolation, a history of mental health issues, severe stress, and the side effects of medications can also play a role in worsening depression.

Parkinson’s and Anxiety

Anxiety is another common mood disorder among people living with Parkinson’s. Like depression, anxiety isn’t just a reaction to your diagnosis but is part of the disease itself, rooted in the changes Parkinson’s causes in brain chemistry. About 40% of people with Parkinson’s also experience anxiety disorders, such as generalized anxiety disorder, social avoidance disorder, or obsessive-compulsive disorder.

These anxiety disorders can significantly impact your quality of life, making it difficult to cope with everyday challenges and potentially worsening the physical symptoms of Parkinson’s.

Parkinson’s and Psychosis

Psychosis, though less common than depression and anxiety, can be one of the most distressing mental health challenges associated with Parkinson’s. It typically manifests as visual hallucinations and illusions, where you might see things that aren’t really there or misinterpret visual information. Severe psychosis—characterized by delusions and significant behavioral disturbances—affects about 5% to 10% of people with Parkinson’s.

How to Manage Anxiety and Depression When Living with Parkinson’s

The goal of management of PD is to control the signs and symptoms for as long as possible while reducing the negative effect.

Counseling

Counseling is a valuable tool in managing the emotional and psychological aspects of Parkinson’s. Your trained therapist who understands your medical history will help you navigate your feelings, develop coping strategies, and find ways to maintain a positive outlook. Therapy provides a safe space to express your concerns and work through the complex emotions that PD can bring.

Medication

Your doctor may also prescribe antidepressants or anti-anxiety medications like SSRIs and SNRIs. These help balance the chemicals in your brain, which can reduce the symptoms of depression and anxiety.

Combining Approaches

The most effective way to manage anxiety and depression in Parkinson’s is often a combination of counseling, medication, and lifestyle changes. Regular physical activity, maintaining social connections, and practicing relaxation techniques can all enhance the benefits of counseling and medication.

• Regular exercise boosts your mood and helps reduce stress. Even simple activities like walking, stretching, or light workouts can make a big difference. You can also join an exercise program tailored for people with Parkinson’s.
• You might also find it helpful to join a support group. Being around others who understand what you’re going through can provide comfort and encouragement. It’s good to know that you’re not alone.
• Parkinson’s can make you feel like you’re losing control, but focusing on what you can control helps reduce anxiety. Establish a routine, set small goals, and celebrate your achievements, no matter how small they seem, while also practicing relaxation techniques to maintain control over your mental health.

Coping with Parkinson’s disease is a journey. Some days are better than others, and that’s okay. Focus on taking one day at a time and be kind to yourself. Remember, managing anxiety and depression is a process, and you don’t have to do it alone.

Science 37 offers a clinical trial specifically for Parkinson's Disease. This trial provides an opportunity for individuals to participate in cutting-edge research that aims to improve the understanding and treatment of the disease. By joining, you can contribute to advancements in medical science while potentially gaining access to new therapies and support tailored to your condition. If you're interested in exploring this option, participating in a clinical trial could be a valuable step in your journey with Parkinson's.

Under normal circumstances, your heart beats in a steady rhythm, usually between 60 and 100 times per minute when you’re resting. This regular beat is what keeps blood flowing smoothly through your body. However, in atrial fibrillation (AFib), the rhythm is off, and the two upper chambers of the heart (the atria) can beat up to 300 times per minute in a chaotic and uncoordinated way. You can usually check this by feeling your pulse at your wrist or neck. 

This irregular heart rhythm makes it difficult for the heart to pump blood effectively. When blood pools in the atria, it increases the risk of forming blood clots. If one of these clots reaches your brain, it can block blood flow and cause an ischemic stroke, which is a stroke caused by a blocked artery. According to experts, AFib is responsible for 15 to 20% of all strokes, many of which lead to heart failure, permanent disability, or death.

Alarming as it is, AFib is not often life-threatening. However, it does require treatment to manage the symptoms and reduce the risk of stroke. Common treatments include medications to prevent stroke and control the heart's rhythm or rate, as well as procedures like cardioversion and catheter ablation. Your doctor may also recommend a left atrial appendage if you can’t take a blood thinner due to the risk of bleeding or falls.

Clinical trials are also underway, testing new treatments and therapies that aim to reduce the risks of AFib, improve patient care, and make managing heart health easier and more effective.

New Advances in AFib Treatment

Medications for atrial fibrillation (AFib) can help manage the condition, but they’re not without their drawbacks. While these drugs are effective, they also come with limitations and can sometimes cause side effects that are uncomfortable or even dangerous. For example, a recent study found that patients on anti-AFib medications often need more pacemakers and face more hospitalizations due to a slower heart rate. Because of these risks, doctors need to carefully weigh the benefits of these medications against their potential downsides.

Thankfully, there are other options beyond medication. One of the most promising alternatives is AFib ablation, also known as catheter ablation. This procedure creates tiny scars in specific areas of the heart to block the abnormal electrical signals that cause arrhythmia.

Studies have shown that a single ablation procedure can reduce or eliminate AFib in about 70% to 80% of patients. This makes ablation more effective than drug therapy in controlling irregular heart rhythms.

However, existing ablative therapy has limitations, too. These include potentially highly morbid complications like phrenic nerve injury and atrial fistula. According to experts, traditional approaches use thermal energy sources like laser, radiofrequency, cryo energy, etc., all of which carry an associated short-and long-term injury risk to nearby structures.

These limitations led to new technology that is even more effective and safer. Let’s explore some of the latest innovations in AFib ablation:

Pulsed Field Ablation

Pulsed Field Ablation (PFA) is a newer technique that uses short, precise bursts of electrical energy to create scar tissue in the heart. The FDA approved the first PFA system in December 2023 to treat persistent and paroxysmal atrial fibrillation. A recent review found PFA to be a promising approach to arrhythmia atrial fibrillation treatment due to its improved safety compared to thermal ablation. PFA only targets heart cells that need to be treated while leaving surrounding tissues unharmed.

Ganglionated Plexi Ablation

A ganglionated plexi ablation is a treatment option for a specific group of AFib patients. Research shows that ganglionated plexi—clusters of nerve cells—are commonly found on the back surface of the right atrium. These nerve clusters are believed to play a key role in triggering and sustaining AFib. Targeting and ablating these nerve clusters in the left atrium provides a more personalized treatment option, especially for patients whose AFib is linked to nerve activity.

Convergent Procedure

The convergent procedure is a hybrid technique that combines the strengths of catheter ablation with a minimally invasive surgical approach. This allows doctors to reach and treat areas of the heart that are difficult to access with traditional methods. It’s a more comprehensive treatment that can improve the chances of successfully managing persistent AFib.

Vein of Marshall Ablation

Vein of Marshall Ablation is a minimally invasive procedure for people who experience persistent atrial fibrillation symptoms. Patients with atrial fibrillation usually start with a pulmonary vein isolation ablation to stop the irregular electrical signals that cause abnormal heart rhythms. However, if the symptoms last longer than seven days, this procedure alone may not be enough.

For those with persistent symptoms, an additional treatment called vein of Marshall Ablation with ethanol infusion can be used. When combined with pulmonary vein isolation, this procedure targets and removes extra triggers and risk factors that can cause atrial fibrillation.

New Medications

Traditionally, anticoagulant therapy is used to reduce the risk of stroke in people with AFib. However, these medications can increase the risk of bleeding, which is a serious concern. New medications being tested in clinical trials for heart diseases aim to offer effective stroke prevention with fewer adverse events. These drugs could become the new standard of care if they prove safer and more effective than existing options.

The Impact of Clinical Trials on Heart Health

Clinical trials are crucial because they help doctors and researchers learn more about what works best in real-world situations. They provide valuable insights into how new treatments can improve patient outcomes, reduce the risk of stroke, and enhance the quality of life for those living with AFib.

As innovations are tested and refined, they have the potential to change the way AFib is treated across the world. And the best part is that you can participate in this process by enrolling in a clinical trial. 

The ongoing research and development in the field of atrial fibrillation treatment offer hope for better, safer, and more effective ways to manage this condition. As clinical trials continue to explore new possibilities, the advancements made today will pave the way for improved heart health tomorrow.

Asthma can be tough, especially when it’s your child or someone you care about struggling to breathe. But even though there’s no cure, asthma doesn’t have to stop you or your loved one from living a normal, happy life. With the right tips and support, you can manage asthma and breathe easier. In this guide, we’ll share practical tips and advice to help you handle asthma day by day.

Understanding Asthma

Asthma is a lung disease. It’s chronic, which means it doesn’t go away and needs ongoing medical management. Asthma is one of the most common non-communicable diseases and a leading chronic disease in children. According to the Asthma and Allergy Foundation of America, over 27 million (1 in 12) US adults ages 18 and older had asthma in 2023. During the same period, about 4.5 million children under 18 had asthma.

Asthma can be a minor nuisance for some. But for others, it can be a major problem that affects daily activities and leads to a life-threatening asthma attack. That’s why it’s critical to seek medical help when you have or suspect that you have asthma. Early treatment may also prevent long-term lung damage. Some common symptoms to watch out for include:

• Shortness of breath
• Chest tightness or pain
• Wheezing when exhaling
• Wheezing or coughing attacks that are worsened by viral infections like flu or cold
• Trouble sleeping

Researchers still don’t know why some people have asthma while others don’t. But environmental factors, genetics, respiratory infections, and allergies are believed to increase the risk. Environmental factors are particularly harmful to infants and young children whose immune systems are still developing.

How to Manage Childhood Asthma

Managing childhood asthma involves taking proactive steps to reduce the risk of asthma attacks and improve your child's quality of life. Some children outgrow asthma as they get older, but it’s crucial to control symptoms and minimize exposure to triggers in the meantime. Let’s explore ways to manage childhood trauma:

Recognize Triggers

Triggers are things that irritate your airways and set off your asthma symptoms. People have different triggers, so you want to know which ones affect you or your loved one. Some common triggers include cigarette smoke, dust mites, mold, pollen, pet dander, physical activity, weather, chemicals, metal salts, certain medicines, high emotions, etc.

Sometimes, it can be obvious what triggers your asthma. If you get symptoms after coming in contact with smoke from open fires or cigarettes, then smoke is probably one of your triggers. The same applies to contact with pets like dogs and cats. However, triggers like pollen or air pollution may be hard to determine because allergens aren’t visible.

You should, therefore, keep a diary of when you experience symptoms, your location, the weather conditions and the things you were exposed to, including stress.

Avoid Triggers

Once you understand your triggers, the next step is to focus your efforts in the right area. Avoiding them at all times might not be possible, but you can minimize exposure so your symptoms are easier to manage. Here are some quick tips on how to go about it:

• Limit exposure to dust mites: Use dust-mite-proof covers on mattresses and pillows, wash bedding in hot water weekly, and keep stuffed animals off the bed.
• Reduce pet dander: If your child is allergic to pets, limit their contact with animals. Keep pets out of your child's bedroom and clean regularly to reduce dander.
• Control hay fever: During allergy season, keep windows closed and use air conditioning to filter the air, which will help reduce pollen exposure.
• Avoid smoke and vape chemicals: Keep your child away from secondhand smoke and vaping areas, as these are major irritants for children with asthma.
• Watch out for strong odors: Avoid using strong cleaning products or cooking spices that might trigger symptoms. Opt for fragrance-free or hypoallergenic options instead.

Maintain a Medication Routine

Asthma is a chronic illness that doesn’t go away and requires ongoing management. Even if your child feels fine, their lungs might still be inflamed. Asthma medication—especially inhaled corticosteroids and other controller medicines—reduces inflammation and keeps airways open. Skipping doses or only using medication when symptoms appear can lead to more frequent and severe asthma attacks.

How to Do It

• Set a schedule: Find a routine that fits into your daily life. Whether it’s right after brushing teeth in the morning or before bedtime, consistency helps ensure your child takes their medication regularly.
• Use reminders: Alarms on your phone or sticky notes on the bathroom mirror can serve as helpful reminders to keep up with the medication schedule.
• Involve your child: As your child grows, help them understand the importance of their medication. Encourage them to take responsibility for their routine to make them more consistent and aware of their health needs.
• Keep medications accessible: Store medications in an easy-to-reach spot that’s also safe from little hands. If your child uses an inhaler, ensure it’s always nearby, especially during sports or outings.
• Review the plan regularly: Check in with your child’s healthcare provider to ensure the current medication routine works. Adjustments may be needed as your child grows or if their symptoms change.

Create an Asthma Action Plan

You’ll need an action plan to manage your child’s asthma effectively. A good asthma action plan outlines what to do daily to keep asthma under control and the steps to take when symptoms worsen. This way, you can recognize early warning signs, know what to do and communicate with everyone involved in your child’s care.

How to Create an Asthma Action Plan

1. Work with your healthcare provider: Collaborate with your child’s doctor to develop a plan tailored to their needs. The doctor will help set up different zones based on how well your child’s asthma is controlled.
2. Define the zones:

• Green zone (good control): This is when your child’s asthma is well-managed. Include information about their daily medications and what to do to maintain control.
• Yellow zone (caution): This indicates that asthma symptoms are worsening. List the steps to take, such as adjusting medications or using a rescue inhaler, and when to contact a healthcare provider.
• Red zone (emergency): This signals a serious asthma episode. Provide instructions for immediate action, including using a rescue inhaler or nebulizer and when to call emergency services or go to the emergency room.

3.    Include clear instructions: Detail how and when to use medications, including dosages and timing. Also, provide guidance on recognizing worsening symptoms and how to respond.

1. Update the plan regularly: As your child grows or their asthma changes, revisit the plan with their healthcare provider. Make updates to ensure it continues to meet their needs.
2. Share the plan: Give copies of the Asthma Action Plan to everyone involved in your child’s care, including school staff, family members, and caregivers. Ensure they understand what to do and where the plan is kept.
3. Practice with your child: Review the plan with your child and practice the steps, especially what to do if they enter the yellow or red zones. This helps them feel more confident and in control of their condition.

Get Regular Check-Ups

Regular check-ups with your child’s healthcare provider are essential to controlling asthma. During these visits, your provider will perform a physical exam to assess your child’s lung function. They will review and adjust medication as necessary and advice on the latest advancements, including clinical trials. Regular appointments are a good time to ask questions and ensure your child’s asthma management plan is still effective.Science 37 is currently conducting a clinical trial for asthma. This trial is designed to explore new treatment options and improve the management of asthma symptoms. By participating, you can help advance medical research and potentially benefit from innovative therapies that could enhance your quality of life. If you're living with asthma and are interested in contributing to scientific progress, this clinical trial could be a valuable opportunity to explore new possibilities for managing your condition.

Fragile X syndrome (FXS) is the most common form of inherited intellectual disability, affecting about 1 in 7,000 males and 1 in 11,000 females worldwide. The disorder causes behavioral issues, physical abnormalities, and a range of other health problems. Currently, there’s no cure for FXS. However, interventions like speech therapy, special education, behavioral and physical therapy, and medication offer the chance for patients to optimize a full range of skills. Ongoing FXS research also paves the way for new treatments that provide hope to those affected by this condition. 

An Overview of Fragile X Syndrome

FXS is a genetic disorder caused by a mutation in the Fragile X Messenger Ribonucleoprotein 1 (FMR1) gene located on the X chromosome. This gene is responsible for producing a protein called FMRP, which helps in brain development and the formation of connections between brain cells vital for learning and cognitive function.

For those with FXS, the changes in the FMR1 gene prevent it from producing sufficient FMRP. This leads to symptoms like learning disabilities, developmental disabilities, and behavioral challenges. People with FXS often struggle with reasoning, problem-solving, and applying knowledge in everyday situations. They may also have social and emotional issues like difficulty understanding social cues, forming relationships, or managing social interactions. Autism spectrum disorder (ASD) is also common among those with FXS.

In addition to behavioral and cognitive impairments, FXS may also present distinct physical features, such as a long face, large ears, and flexible joints. These features, along with other issues like flat feet and low muscle tone, can affect physical coordination and mobility. These symptoms are not life-threatening, and those with the condition typically can expect a normal life expectancy.

While some people with Fragile X-associated disorders may still produce some FMRP, the severity of symptoms is primarily determined by how much of the protein is produced. Males who have FXS often have some level of intellectual disability that ranges from mild to severe. On the other hand, females may have some level of intellectual disability or normal intelligence.

Risk Factors

FXS is an inherited genetic disorder. However, several specific risk factors can increase the likelihood of a person being affected by or carrying the mutation. These include carrier parents, one or more family members affected by the condition, etc. 

Emerging Trends and Future Advancements in Treatment

Fragile X syndrome has no cure at the moment. But there are off-label drugs that target specific symptoms of the disease, such as SSRIs for anxiety and depressive symptoms, and stimulants that include methylphenidate for impulsivity, inattention, and hyperactivity. People with FXS may also get behavioral health counseling and therapy to address the psychological aspects of the condition. Education, medication, and therapy services give FXS patients the best chance to use all their skills and capabilities. 

But there are ongoing studies and clinical trials to try to find FSX treatment. Let’s explore some of the emerging research and latest advancements in the field:

Gene Therapy & Protein Replacement Studies

One of the most exciting areas of research for Fragile X Syndrome is gene therapy. Scientists are exploring ways to fix FXS at its root cause by reactivating the silenced FMR1 gene. Technologies like CRISPR gene therapy are being used to restore the function of this crucial gene in people with the full mutation.

Such groundbreaking studies show promising results, offering hope that this approach might lead to a viable treatment. While more work is needed before these techniques are ready for clinical use, the progress made so far is promising.

Correcting Genetic Processing Errors

Another promising development comes from recent discoveries about the FMR1 gene. Contrary to earlier beliefs, researchers found that the mutated gene responsible for FXS is active in most people (about 70%) with the disorder. However, the gene fails to produce the necessary FMRP protein because the genetic material isn’t processed correctly.

Repositioning Treatments in Development

In addition to gene therapy and genetic processing corrections, various therapies, and research strategies are being explored for FXS. Researchers are investigating how these therapies might target the symptoms and underlying mechanisms of FXS. 

Clinical Trials

Clinical trials provide the ultimate confirmation that a therapy is safe and effective for use in humans. They play a vital role in translating promising research into real-world solutions for those affected by Fragile X Syndrome (FXS). Many organizations and research institutions are conducting clinical trials to evaluate new and innovative treatments for FXS, offering hope to individuals and families impacted by the disorder.

Science 37 is taking the lead with FXS clinical trials. As a pioneering clinical trial technology company, we’re changing how clinical trials are conducted for Fragile X Syndrome and other conditions. Our innovative approach focuses on creating more accessible and patient-centric trials.

We employ advanced digital platforms that allow clinical trials to be conducted virtually, a method known as decentralized clinical trials (DCTs). This approach enables participants to participate in trials from the comfort of their homes, reducing the need to travel to specialized medical centers. For families dealing with the challenges of FXS, this model offers significant advantages like:

• Increased accessibility
• Streamlined processes
• Patient-centric design

Several ongoing clinical trials explore novel treatments for FXS, leveraging the advantages of Science 37's technology. From gene therapy trials to targeted molecular treatments to behavioral and cognitive interventions, these trials pave the way for potentially transformative therapies for FXS.

Your liver is an essential organ that performs many crucial functions to keep your body healthy. It processes nutrients from your food, detoxifies harmful substances, produces bile to aid digestion, and helps with blood clotting. Essentially, the liver keeps your body's metabolism running smoothly.

But conditions like primary biliary cirrhosis (PBC) and primary sclerosing cholangitis (PSC) disrupt these functions. Each condition has its unique features and progresses differently, but both may eventually lead to cirrhosis and liver failure. In this guide, we’ll explore how PBC and PSC affect liver function and the mechanisms by which they damage the bile ducts and liver cells. 

What's Primary Biliary Cirrhosis? 

PBC or primary biliary cholangitis is an autoimmune disease that primarily affects the bile ducts within the liver. So basically, your immune system mistakenly attacks the bile ducts, leading to inflammation and scarring. Over time, this degrades the ducts and makes it harder for bile to flow to your small intestine.

As a result, bile backs up inside your liver and damages the tissue. Scar tissue slowly replaces healthy tissue, and your liver eventually loses its functionality. PBC is chronic and progressive, meaning it doesn’t go away and worsens over time.

Progression and Impact on the Liver

PBC progresses slowly through several stages. In the beginning, it’s mild, and you might miss it. But in the end, the liver becomes increasingly scarred, leading to a condition known as cirrhosis. Cirrhosis marks the end-stage of liver disease, where liver function is significantly impaired. If left untreated, this can eventually cause liver failure, which is life-threatening.

The progression of PBC varies from person to person, but understanding its impact is essential for managing the disease. Primary Biliary Cirrhosis symptoms include itching, dry eyes and mouth, and jaundice.

What is Primary Sclerosing Cholangitis?

PSC is another chronic liver disease that affects the bile ducts. However, PSC tends to cause inflammation and scarring in both the small and large bile ducts inside and outside the liver. This differs from PBC, which primarily affects the smaller bile ducts within the liver.

The exact cause of PSC is not well understood, but it's believed to be linked to genetic and environmental factors. Like PBC, PSC is also considered an autoimmune disease. It’s worth noting that PSC often accompanies inflammatory bowel disease, particularly ulcerative colitis, hinting at a complex relationship between the liver and immune system.

Mechanism of PSC

In PSC, the inflammation causes scarring and narrowing of the bile ducts. This blockage impedes bile flow, leading to liver cell damage. The obstruction of bile acids results in liver inflammation, contributing to the progression of liver disease.

Progression and Impact on the Liver

The disease progresses slowly in most people with PSC and causes symptoms like fatigue, abdominal pain, itchy skin, and jaundice. Eventually, you may experience repeated infections, portal hypertension, metabolic diseases, tumors, or liver or duct and liver failure. At the moment, the only known cure for advanced PSC is a liver transplant. However, the disease may recur in the transplanted liver in a small number of patients.

The Role of Bile Acids and Liver Dysfunction

Bile acids play a vital role in digestion, helping to break down fats and absorb fat-soluble vitamins. In both PBC and PSC, the inflammation and scarring of bile ducts can obstruct bile flow, causing bile acids to accumulate in the liver and bloodstream.

This buildup can be toxic to liver cells and contributes to liver disease progression. It can also lead to digestive issues, affecting your body's ability to process nutrients effectively. When bile flow is impaired, it can also impact the small intestine, leading to nutritional deficiencies and digestive discomfort. This disruption in digestion highlights the importance of maintaining healthy bile flow for overall health.

Similarities and Differences Between PBC and PSC

PSC and PBC are both chronic liver diseases that primarily affect the bile ducts. Both diseases are progressive and are thought to have an autoimmune component, where the body’s immune system mistakenly attacks the bile ducts. The attack causes inflammation and subsequent damage. PBC and PSC also cause similar symptoms and side effects like: 

• Fatigue: A feeling of constant tiredness and lack of energy.
• Itching (Pruritus): A common symptom caused by the buildup of bile acids in the bloodstream.
• Jaundice: Yellowing of the skin and eyes due to increased bilirubin levels.
• Abdominal Discomfort: Pain or discomfort in the upper right abdomen.

But the two are also different in the following ways: 

Disease Mechanism

PBC primarily affects the intrahepatic (or small) bile ducts within the liver. In contrast, PSC affects both the intrahepatic (inside the liver) and extrahepatic (outside the liver) bile ducts. While you can treat PBC with medications, PSC is more complicated and has no effective treatment.

Affected Population

PBC is more common in middle-aged women, with about 90%of cases occurring in females. This suggests a potential hormonal or genetic link in its development. On the other hand, PSC tends to affect young to middle-aged men more frequently, although it can occur in both genders. 

Associated Conditions

PBC is not typically associated with other diseases, although some patients may have other autoimmune conditions, such as rheumatoid arthritis or Sjögren's syndrome. PSC, however, has a strong link with inflammatory bowel disease (IBD), particularly ulcerative colitis or Crohn’s disease. Up to 80% of PSC patients may have IBD. 

Diagnostic Markers

PBC is often diagnosed through blood tests that detect specific antibodies, such as anti-mitochondrial antibodies (AMA) or anti-nuclear antibodies (ANA). These antibodies are present in about 95% of PBC patients and provide a clear marker for diagnosis, making it easier for doctors to identify PBC early. 

There are no specific antibodies in PSC like in PBC, making diagnosis more challenging. Instead, PSC is typically diagnosed through imaging studies like MRCP (magnetic resonance cholangiopancreatography) or ERCP (endoscopic retrograde cholangiopancreatography) that reveal characteristic bile duct changes. Liver biopsy may also be used to assess the extent of liver damage.

Treatment

PBC treatment mainly focuses on managing signs and symptoms and slowing disease progression with medications like ursodeoxycholic acid (UDCA), which helps improve bile flow and reduce liver inflammation. Newer medications are also being explored to offer more targeted therapies for PBC patients.

PSC lacks a specific medical treatment. Treatment often involves managing complications and considering liver transplantation for advanced cases. Endoscopic procedures may be used to manage bile duct strictures, but long-term management still remains a challenge.

Organizations like Science 37 are running trials for new treatments or improving existing ones. If you need more detailed information on clinical trials or specific aspects of treatment, please feel free to ask.

Most people who struggle with mental health disorders have sleep troubles, and vice versa. This is because the two are closely connected, oftentimes with one leading to the other. Studies have linked sleep disorders like obstructive sleep apnea, restless legs, and narcolepsy to depression, anxiety, bipolar and other mental health disorders. At the same time, these mental health issues may also cause poor sleep. 

What Is Narcolepsy?

Narcolepsy is a chronic sleep disorder with no known cause. It affects the brain's ability to control sleep-wake cycles, resulting in excessive daytime sleepiness. Under normal circumstances, the sleep-wake cycle includes various stages that gradually transition from light sleep to deep sleep and finally to REM (Rapid Eye Movement) sleep. 

REM sleep is essential for dreaming and helps rejuvenate your mind and body. However, if you have narcolepsy, this process is disrupted. Instead of following the typical progression through the sleep stages, you quickly enter REM sleep shortly after falling asleep, skipping the initial stages.

You’ll also experience fleeting urges to sleep. When the urge becomes overwhelming, you’ll fall asleep for a few seconds to several minutes – though sometimes, you may remain asleep for an hour or longer.

Symptoms of Narcolepsy

Here are some common narcolepsy symptoms:

1. Cataplexy, sudden muscle weakness
2. Excessive daytime sleepiness (EDS)
3. Sleep paralysis
4. Hypnagogic hallucinations
5. Disrupted nighttime sleep and waking up often
6. Continual lack of energy and intense fatigue
7. Depression
8. Weak limbs
9. Vision problems
10. Difficulty in focusing and memorizing 
11. Automatic behavior, where you perform routine tasks without conscious awareness of doing so

Narcolepsy and Mental Health 

Narcolepsy often co-occurs with mental health issues like depression and anxiety. 

Depression and Anxiety

According to a survey, about 57% of narcolepsy patients said they’re depressed. In contrast, only about 4.7% of adults in the US say they have regular feelings of depression.  At the same time, about 35% of people with narcolepsy had anxiety disorders like social phobias and panic attacks. That compares to about 18% of the general population.

ADHD

Scientists believe that there’s a link between attention deficit hyperactivity disorder and narcolepsy. Although the two medical conditions seem to be on opposite ends because of how they present themselves, a study found that children with narcolepsy are more likely to present ADHA symptoms than those with regular sleep patterns.

Eating Disorders

People with narcolepsy are also at a higher risk for developing eating disorders, especially binge eating and abnormal food cravings. In one study, about a quarter of those with narcolepsy met the criteria for having an eating disorder.

Schizophrenia

Narcolepsy and schizophrenia share some similar symptoms, such as hallucinations. Researchers have looked into cases where individuals show signs of both disorders. These issues often begin in childhood or the teenage years. However, being diagnosed with both schizophrenia and narcolepsy is relatively rare.

How Narcolepsy Fuels Emotional Distress

The unpredictable nature of narcolepsy often makes mental health issues like depression and anxiety even harder to manage. For example:

• The sudden onset of sleep attacks (where one falls asleep unexpectedly while having a conversation, at work, or during a social event) is not just embarrassing but can also increase worry and anxiety. Narcolepsy symptoms can also cause work and relationship issues. 
• Cataplexy also makes it difficult to fully experience emotions. A strong emotion like surprise or laughter may lead to an unexpected muscle weakness. 
• Sleep paralysis and hallucinations don’t make it any easier. These experiences can be frightening and contribute to anxiety and fear around sleeping. You might find yourself dreading bedtime, leading to disrupted sleep and worsening narcolepsy symptoms.
• And when you don’t sleep well, the hormones that regulate your appetite may be substantially lowered, which can contribute to the development of eating disorders.

Many people are likely to isolate themselves, due to the worry or fear of these unpredictable symptoms. You might avoid gatherings, work events, or outings with friends to prevent potential embarrassment. But isolation isn’t good for your mental health. In fact, according to studies, feelings of isolation are linked to suicidal thoughts, less restful sleep, more stress, and a decrease in your ability to regulate eating habits.

The unpredictable nature of this sleep disorder can also chip away at your self-esteem. The sudden symptoms might make you feel inadequate – especially since you struggle to maintain a routine or keep up with responsibilities. Low self-esteem is known to cause persistent feelings of sadness, depression, anger, anxiety, guilt, or shame.

Sadly, mental issues like anxiety or depression make narcolepsy symptoms worse. High-stress levels can disrupt your sleep patterns, leading to more daytime sleepiness and sleep attacks. It’s a vicious cycle where poor sleep increases emotional distress, and emotional distress worsens sleep quality. The more stressed and anxious you feel, the harder it becomes to get restorative sleep, which is crucial for managing narcolepsy.

Ways to Improve Narcolepsy and Sleep

Both sleep and mental health can have a major impact on quality of life, so it’s crucial to get proper care that involves working with a trained health professional. Your doctor or psychiatrist will assess the benefits and risks of different types of treatments and offer customized care even in situations with multiple co-occurring mental or physical issues.

One of the go-to treatments for narcolepsy is sodium oxybate. This prescription drug is given for narcolepsy with cataplexy and excessive daytime sleepiness. It helps regulate your sleep patterns so you get better rest at night and feel more awake during the day.

Sodium oxybate targets specific brain chemicals that keep your sleep-wake cycles more stable. Other medications, like antidepressants and stimulants, help to improve your mood and sleep, and can boost your energy and alertness.

Beyond medications, your doctor may recommend therapy for both conditions. Behavioral therapies like Cognitive Behavioral Therapy (CBT) can help reframe your thinking. Your therapist will use different types of CBT for specific problems like bipolar disorder, anxiety, and depression.

They may also use CBT for insomnia (CBT-I) to help reduce sleep problems. A large clinical research study showed that CBT-I can lessen the symptoms of many mental health conditions.

You may also need to make simple lifestyle changes to manage the conditions. Try to stick to a regular sleep schedule, get some light exercise, and practice good sleep habits. These can all help you feel better during the day. Relaxation techniques like meditation or yoga are great for reducing stress and boosting your immune system.

Exciting things are happening in the world of narcolepsy research. Plenty of clinical trials are looking into new treatments. For example, Science 37 is working on some cool studies to discover new ways to tackle narcolepsy. These trials aim to find more effective treatments that can offer real relief and improve the quality of life for people with narcolepsy.

Sickle Cell Disease (SCD) is a genetic blood disorder that affects the shape and function of red blood cells. Instead of being round and flexible, like healthy red blood cells, the cells in someone with SCD are crescent or "sickle" shaped. The change in shape causes these cells to get stuck in blood vessels, leading to painful episodes known as crises and other serious health issues like stroke, eye problems, infections, chronic pain, chest pain, and shortness of breath. 

Living with SCD is tough, but the proper nutrition can help. The right foods can help your body produce healthier red blood cells, reduce pain and inflammation, and give you the energy you need to feel your best. 

Understanding Sickle Cell Disease

SCD is a genetic blood disorder that’s marked by flawed hemoglobin (hb). Hb is the protein in the red blood cells that transports oxygen to the body tissues. SCD interferes with the delivery of oxygen to the tissues. 

What Causes Sickle Cell Disease?

SCD is inherited in an autosomal recessive pattern, meaning a person must inherit two abnormal hemoglobin genes (one from each parent) to develop the disease. If someone inherits one abnormal gene and one normal gene, they will have sickle cell trait, which typically doesn't cause symptoms of SCD but can be passed on to offspring. Risk factors include: 

• Family history of sickle cell disease
• People who are African Americans or from Middle Eastern, Mediterranean, and Indian ancestry
• Genetic inheritance

Signs and Symptoms of Sickle Cell Disease

Signs and symptoms of SCD can include:

• Anemia
• Acute chest syndrome
• Pain crisis (sickle crisis)
• Splenic sequestration (pooling)
• Jaundice
• Priapism

Please note that these symptoms vary in intensity and require prompt medical attention when severe. Symptoms like acute chest syndrome, where sickle cells block vessels in the lungs and cause chest pain, fever, and breathing difficulty, can be life-threatening. So, it’s critical to work closely with your healthcare provider to monitor and address these symptoms as they arise.

SCD Diagnosis 

Diagnosis is done early in life with a newborn screening. This test checks for the presence of sickle hemoglobin shortly after birth. Early diagnosis is super important because it lets doctors start treatment right away to prevent complications. If you are pregnant and suspect that you, your partner, or both of you have the sickle cell trait, genetic counseling can help clarify the situation and provide guidance on the potential risks for your child.

Sickle Cell Disease Treatment

SCD is a lifelong disease. Most treatments are designed to help improve quality of life, though options like bone marrow transfer have been shown to potentially cure SCD: 

• Pain management, which involves the use of medications to relieve pain during sickle cell crises.
• Hydroxyurea, a medication that helps reduce the frequency of pain episodes and other complications.
• Blood transfusions, to help increase healthy red blood cells and prevent complications like stroke.
• Bone marrow transplant, which involves replacing diseased bone marrow with healthy marrow from a donor.  

Foods that Help Improve Health in Sickle Cell Diseas

A healthy diet supplies the body with the nutrients it needs to fight infections and promote health. Here are some great goods to integrate as part of your diet when you have sickle cell disease:

Fluids

Hydration is good for the body, even under normal circumstances. But it’s critical when you have sickle cell. Your body needs fluids to keep the blood flowing smoothly and prevent sickle cells from clumping together. Go for at least eight glasses of water a day. Herbal teas and natural fruit juices without added sugars are also great choices.

Iron-Rich Foods

Iron is super important because it helps your body make healthy red blood cells, which helps fight anemia – a common issue with SCD. Try to include the following foods and pair them with foods rich in Vitamin C (bell peppers or oranges) for better absorption.

• Fish: Especially sardines and salmon
• Leafy greens: Like spinach, kale, and Swiss chard
• Lean meats: Like beef, chicken, and turkey
• Legumes: Including lentils, beans, and chickpeas

Folate-Rich Foods

Folate also does a great job helping the body produce red blood cells. Great examples of folate-rich foods are: 

• Leafy greens: Spinach, collard greens, broccoli, and romaine lettuce
• Citrus fruits: Oranges, lemons, and grapefruits
• Beans and peas: Black-eyed peas and kidney beans
• Fortified cereals and breads

Antioxidant-Rich Foods

Such foods help reduce oxidative stress and inflammation, which is beneficial for SCD. These foods, rich antioxidants, can help protect your cells and support your immune system:

• Berries: Blueberries, strawberries, and raspberries
• Seeds and nuts: Almonds, walnuts, and sunflower seeds
• Colorful vegetables: Including carrots, bell peppers, and broccoli
• Green tea

Omega-3 Fatty Acids

These foods have anti-inflammatory properties that can help reduce pain and improve your overall health. They also are good for your heart health. Foods rich in omega-3s include:

• Fatty fish: Like salmon, mackerel, and sardines
• Flaxseeds and chia seeds
• Walnuts
• Soybeans

Protein-Rich Foods

Proteins help your body repair and grow, which is great for SCD. Make sure to get a variety of protein sources:

• Lean meats and poultry: Chicken and turkey are good options.
• Fish and seafood: Rich in protein and often in omega-3s.
• Eggs: A versatile and complete protein source.
• Dairy products: Milk, cheese, and yogurt provide both protein and calcium.
• Plant-based proteins: Tofu, tempeh, and edamame are excellent for variety. Plant-based proteins: Including tofu, tempeh, and edamame

Foods to Avoid if You Have Sickle Cell Disease

Just as there are foods that can help you feel better with sickle cell disease, there are also foods that might make your symptoms worse. Here are some types of foods you should try to avoid:

High-Sodium Foods

Foods high in sodium can increase your blood pressure and cause your body to hold onto extra fluids. This puts more strain on your heart and blood vessels. Lower your sodium intake to help keep your heart and blood vessels healthy.

Gluten

Foods rich in gluten tend to worsen anemia. Gluten may damage the intestinal wall and limit folic and iron absorption, which are essential in the production of red blood cells.

High-Fat Foods

Unhealthy fats, especially saturated and trans fats, cause inflammation and increase the risk of heart disease. It's important to maintain a healthy weight and manage your cholesterol levels to keep your heart in good shape.

Caffeine and Alcohol

Limit or avoid caffeine and alcohol intake. Caffeine contains tannins which bind to iron molecules and interfere with iron absorption in the body. Alcohol affects the production and maturation of red blood cells, causing abnormality or dysfunction of cells. It also affects how nutrients are absorbed from food and may lead to folic and iron deficiencies. Until recently, a bone marrow transplant was the only cure. But in December 2023, two new therapies got FDA approval to treat sickle cell disease. One approach involves adding a gene to the body, while the other modifies an existing gene. Clinical trials are also exploring gene-based treatments alongside other therapies to help reduce symptoms and extend lifespan.

Vaccines protect us from infectious diseases that could otherwise cause illness or even death. They work by training our immune system to recognize and fight off harmful viruses and bacteria before they take hold and cause disease. Vaccines have eradicated or significantly reduced many deadly diseases, like smallpox, polio, RSV flu, and COVID-19, saving millions of lives. But how do vaccines go from concept to being a critical part of our healthcare system?

Introducing a new vaccine to the market involves extensive planning and a series of rigorous steps. The vaccine development process includes many stages, with clinical trials playing a crucial role. These trials are conducted to evaluate the novel vaccine's effectiveness and safety using a specific group of human participants. Let's explore the journey of vaccine clinical trials.

How Are Vaccines Made?

Since the outbreak of the COVID-19 pandemic, we’ve seen an unmatched level of funding for vaccine research and development (R&D). The US Department of Health And Human Services, for instance, awarded more than $1.4b for Project NextGen to support the development of a new generation of technologies and tools to protect against COVID-19. But R&D is only the first of many steps:

Research and Discovery

The journey of a vaccine begins with research and discovery, where scientists identify potential antigens that can trigger an immune response, like the spike protein in the SARS-CoV-2 virus for COVID-19 vaccines. This phase involves extensive laboratory work, experimenting with different formulations to stimulate the immune system safely and effectively.

Researchers collaborate across various fields, conducting numerous experiments to understand immune interactions with these antigens. This foundational stage often takes about 10 to 15 years and aims to develop a formulation that trains the immune system to recognize and combat the disease-causing pathogen.

Preclinical Research 

The vaccine then undergoes preclinical research to prove its concept. This involves using small animal models, such as mice, to study how the vaccine interacts with the immune system. The proof of concept stage helps researchers understand how the vaccine works and identify any potential side effects.

They may also adjust the vaccine formulation to enhance its effectiveness. If the results from these animal studies are promising, demonstrating a strong and safe immune response, the vaccine candidate moves to the next step: clinical trials in humans.

Testing the Vaccine

Once a vaccine candidate shows promise in preclinical research, it enters the clinical trial phase, which involves several stages of testing in humans to ensure safety and efficacy. 

Phase 1

In Phase 1, the vaccine is administered to a small group of 20-100 healthy volunteers. This phase focuses on assessing the vaccine’s safety and determining the appropriate dosage. Researchers monitor participants closely for any side effects and evaluate how well the vaccine induces an immune response.

Phase 2

If Phase 1 results are positive, the vaccine progresses to Phase 2, involving several hundred (usually 100 to 300) participants. This phase expands the safety assessments and begins to evaluate the vaccine's efficacy in a broader population. Participants are often chosen to represent the demographics of the intended recipients, including different ages, health statuses, and backgrounds. The phase offers additional safety information on common side effects or risks and how well the vaccine triggers an immune response.

Phase 3

Phase 3 trials involve thousands (1,000 to 3,000) of participants and aim to confirm the vaccine's effectiveness and monitor for rare side effects. This phase provides a comprehensive assessment of the vaccine's ability to prevent the disease in a real-world setting. The data collected during this phase are critical for the FDA's approval process, ensuring the vaccine meets rigorous standards for public health use.

Phase 4 (After FDA Approval)

After the Food and Drug Administration approves a vaccine, it may enter Phase 4, which involves ongoing studies to monitor long-term safety and efficacy in larger populations. This phase ensures continuous assessment of the vaccine's performance and helps identify any rare or long-term side effects. The Centers for Disease Control and Prevention and other public health organizations play a vital role in this ongoing surveillance, ensuring vaccines remain safe and effective for widespread use.

Manufacturing

Simultaneously with Phase 3 trials, the vaccine's manufacturing process undergoes rigorous scrutiny. The FDA inspects manufacturing facilities to ensure they meet strict quality standards. Each batch of vaccines, known as "lots," undergoes extensive testing to verify consistency and potency. This meticulous process ensures that every vaccine dose produced is safe and effective.

Approving the Vaccine

Before a vaccine can be approved for public use, the manufacturer submits a Biological License Application (BLA) to the FDA. This application includes comprehensive data from preclinical and clinical trials, details of the manufacturing process, and facility inspections. The FDA reviews this data meticulously to determine if the vaccine meets safety and efficacy standards. Approval signifies that the vaccine has undergone rigorous testing and is deemed safe for widespread use.

Recommending the Vaccine for Use

Once approved by the FDA, independent advisory committees, like the Advisory Committee on Immunization Practices (ACIP), review the vaccine's data. They provide recommendations on how, where, and when the vaccine should be administered. Their guidance ensures that vaccines are used effectively to protect public health and address specific population needs.

Monitoring Safety After Approval

Even after a vaccine is approved and distributed, monitoring continues. Systems like the Vaccine Adverse Event Reporting System (VAERS) track potential side effects reported by healthcare providers and patients. Ongoing surveillance ensures swift identification and response to any safety concerns that may arise post-vaccine approval. This continual monitoring is crucial for maintaining public confidence in vaccine safety and effectiveness.

Classification of Vaccines

Vaccines come in two main types: live attenuated vaccines and inactivated vaccines. Both types help protect against serious diseases and keep us healthy.

Live Attenuated Vaccines

Live attenuated vaccines use weakened forms of the actual virus or bacteria. These are strong enough to train the immune system but not cause serious illness in healthy people.

Live attenuated vaccines that are available and routinely recommended in the United States are MMR, rotavirus, varicella, and influenza (intranasal). It may also be used as a typhoid vaccine, adenovirus vaccine, and BCG. 

Inactivated Vaccines

Inactivated vaccines use viruses or bacteria that have been killed. They can’t cause disease but still trigger an immune response. These often need multiple doses and include subunit vaccines (like pneumococcal and influenza vaccines), whole cell inactivated vaccines (like polio, hepatitis A, and rabies), recombinant DNA vaccines (like HPV and hepatitis B), and influenza.

Science 37 is at the forefront of vaccine clinical trials, offering remote participation options that allow you to take part from the comfort of your home. The organization connects researchers, healthcare providers, and participants, making the trial process more efficient and inclusive. By enabling remote involvement, Science 37 accelerates the development of new vaccines, ensuring they are safe and effective for diverse populations. Participants may also be compensated for their time, adding an extra incentive to contribute to this crucial research.

Gout is a really painful type of arthritis that can turn your life upside down. It happens when there's too much uric acid in your blood, which forms sharp crystals in your joints. Imagine waking up in the middle of the night with your big toe feeling like its on fire—red, swollen, and so tender that even the weight of a blanket is unbearable. That’s what a gout attack feels like.

But it's not just about the pain. Gout can make it hard to move around and do everyday things. Walking, exercising, or even just standing may become a challenge. These attacks can come out of nowhere and stick around for days or even weeks. Over time, if gout isn't managed well, it can lead to more frequent attacks and other complications. Gout treatment usually involves a combination of symptom management and lifestyle changes.

What is Gout?

Gout is a form of arthritis that results from an excess of uric acid in the blood. When the body produces too much uric acid or fails to eliminate enough of it, urate crystals can form in the joints, causing intense pain and inflammation. Gout mostly affects the big toe, but can also impact:

• Ankles
• Knees
• Elbows
• Hands and Wrists
• Feet

What Causes Gout in Feet and Other Body Parts?

Gout mostly happens due to high levels of uric acid in the blood. This results from eating foods high in purines, which the body breaks down into uric acid. Purine-rich foods include red meat, organ meats, and certain seafood. High fructose corn syrup, found in sugary drinks and processed foods, also contributes to increased uric acid levels.

Who Gets Gout?

Gout can affect anyone, but it's more common in men than women. Men typically develop gout from ages 30-50, while women are more likely to develop it after menopause. Other groups at risk include those with a family history of gout, individuals with obesity, and people with conditions like high blood pressure, chronic kidney disease, and heart disease. Certain medications like ACE inhibitors, beta-blockers, and anti-rejection drugs are also risk factors.

Symptoms of Gout

Gout symptoms often appear suddenly and can be extremely painful. During an attack, you may experience the following early stage gout symptoms in the affected joint:

• Intense joint pain, which is likely to be severe within the first 4-12 hours after it starts
• Lingering discomfort after the severe pain subsides. This discomfort may last a few days to a few weeks
• Inflammation and redness – the affected joint becomes warm, red, tender, and swollen
• As gout progresses, you may be unable to move your joints normally

People with gout may develop more severe conditions, like recurrent gout, which occurs several times each year. When untreated, gout may lead to the formation of urate crystals under the skin in tophi. Tophi aren’t painful but can become tender and swollen during an attack. The urate crystals may also build up in the urinary tract, leading to kidney stones.

Gout Diagnosis

Gout diagnosis is straightforward. It involves a thorough medical history, physical examination, and specific tests. Your doctor will ask about symptoms and the risk factors we discussed earlier. They will then perform a range of tests to confirm the diagnosis. They may conduct:

• Blood tests to measure your urate levels
• Synovial fluid test, which involves taking a sample of synovial fluid from joints to check for urate crystals
• Scans like x-rays, CT-scan, ultrasounds, or MRIs to spot any joint damage

Gout Management and Treatment

Unfortunately, there’s no cure for gout. However, with the right treatment and lifestyle changes, it can be managed and controlled.

Medications for Treating Gout

Gout treatments are often successful. There are two main parts to treating the conditions:

• Treating the acute attack
• Treatments to prevent future attacks

Treating a Gout Attack

These treatments help you manage the gout symptoms when you experience an attack. The most common medications include:

NSAIDs (Nonsteroidal Anti-Inflammatory Drugs)

NSAIDs Over-the-counter NSAIDs like ibuprofen (Advil, Motrin), diclofenac, and naproxen (Aleve) can help reduce swelling and pain during a gout attack. You take NSAIDs immediately you notice the signs of an attack as this improves the outcome. But NSAIDs aren’t suitable for everyone, so it’s best to talk to your doctor first. Also, you shouldn’t take them for long as they can sometimes cause stomach issues or, with long-term use, more serious problems like ulcers.

Colchicine

Colchicine is a prescription medication that helps reduce pain and inflammation. It’s especially effective if you take it within 24 hours of an attack. The good thing about colchicine is that it specifically targets gout-related inflammation. However, it can cause some stomach upset, so make sure to follow your doctor's dosage instructions.

Corticosteroids

If NSAIDs and colchicine aren’t an option for you, corticosteroids might be the answer. These can be taken orally or injected directly into the affected joint. They’re super effective at reducing inflammation quickly. However, they come with their own set of potential side effects like increased blood sugar, mood swings, and weight gain, so they’re usually reserved for short-term use.

Treatments to Prevent Gout Attacks

Your doctor might prescribe medications (in personalized doses) to help lower your uric acid levels, dissolve away crystals in your joints, and prevent crystals from forming. These are referred to as urate-lowering therapies (ULT). ULT treatments will start once an attack is completely gone.

It might take a few months or even years for the drugs to get rid of the urate crystals from your body. But once they’re gone, you won’t have tophi, gout attacks, or risk of joint damage due to gout. Also, note that these drugs won’t prevent attacks right away. You might still experience attacks up to 6 months after starting them.

 Some common Food and Drug Administration approved ULTs include:

• Xanthine Oxidase Inhibitors (Allopurinol and Febuxostat)
• Uricosurics (Probenecid)
• Pegloticase

Treatment for Joint Damage

When gout leads to joint damage, you’ll likely receive treatment similar to osteoarthritis, which includes:

• Taking painkillers
• Staying at a healthy weight
• Minimizing the strain on your affected joins
• Regular exercise
• Joint replacement surgery in severe cases

Understanding Tophaceous Gout

Tophaceous gout is a particularly severe form of gout that can significantly impact your quality of life. It involves the buildup of large uric acid crystal deposits, known as tophi, which cause chronic joint pain and damage. This condition arises from prolonged, untreated high levels of uric acid in the blood, known as hyperuricemia. Factors like genetics, a diet rich in purines (found in red meat, seafood, and alcohol), obesity, and health conditions such as hypertension, diabetes, and kidney disease increase the risk. Certain medications, including diuretics and aspirin, can also contribute.

The symptoms of tophaceous gout include visible tophi in areas like the fingers, toes, elbows, and ears, leading to joint pain, inflammation, and reduced mobility. If left untreated, it can result in joint destruction, deformity, kidney stones, and kidney damage.

Diagnosis involves a clinical assessment, imaging techniques like X-rays or CT scans, and blood tests for uric acid levels. Treatment aims to relieve pain and lower uric acid levels using medications such as NSAIDs, corticosteroids, colchicine, allopurinol, or febuxostat. Lifestyle changes, including a healthy diet, weight management, staying hydrated, and limiting alcohol intake, are crucial.

Managing tophaceous gout requires a comprehensive approach with medication adherence, regular medical check-ups, and education on self-care practices. Early and effective treatment can prevent complications and improve your quality of life.

Lifestyle Changes to Manage Gout

In addition to the medications, some lifestyle changes can help make a big difference. Here are a few suggestions:

• Try to cut down on red meat, organ meats, and certain seafood like sardines and anchovies, which are high in purines.
• Drinks with high fructose corn syrup, like sodas and beer, can trigger gout attacks. Stick to water, coffee, and tea instead
• Eat more fruits and veggies, especially cherries and berries, which might help lower uric acid levels
• Aim for at least eight glasses of water a day. Staying hydrated helps your kidneys flush out uric acid
• Exercise regularly, but gentle activities like walking, swimming, or cycling are great. High-impact exercises might stress your joints
• Cut back on alcohol
• Reduce stress as it can trigger gout attacks
• You can also do the 10 minute gout cure where you apply cold ice to the joint

What’s the Future for Gout Treatment?

At the moment, gout has no cure. However, clinical studies are underway to explore new treatments and improve existing ones. Researchers are investigating various approaches to managing gout better and possibly curing it, focusing on more effective medications, genetic factors, and lifestyle interventions.

IgA nephropathy, also called Berger’s disease, happens when a protein called immunoglobulin A (IgA) that helps the body fight infections builds up in the kidneys. This buildup causes inflammation and can eventually affect the kidney’s ability to properly filter waste from your blood.

Many people with IgA nephropathy might not realize they have it because it usually doesn’t cause symptoms in the early stages. It can take ten years or more before signs show up, so it’s often called a silent disease.

While there is no cure for IgA nephropathy, there are medications that can help manage the symptoms and sometimes even put the disease into remission.

What Are the Symptoms of IgA Nephropathy?

The disease may not show any symptoms for at least ten years. When symptoms appear, they may include:

• Blood in urine 
• Tea-or cola-colored urine due to the presence of blood. This color may change after a sore throat, cold, or other respiratory infections.
• Edema – swelling of the feet and hands
• High blood pressure
• The urine produces foam
• Fatigue and weakness
• Pain under ribs on both sides of the back

If there’s acute kidney failure, these symptoms may appear:

• Suppressed appetite
• Itchy skin with rashes
• Muscle numbness
• Confusion
• Upset stomach
• Vomiting
• Bitter taste in the mouth

The Causes and Risk Factors of IgA Nephropathy

IgA is a protein known as an antibody that helps fight infections. IgA nephropathy happens when too much of this protein builds up in the kidneys. This buildup occurs in the tiny blood vessels in the kidneys, called glomeruli, causing them to become inflamed.

Scientists still do not know exactly why IgA accumulates in the kidneys. However, certain factors can make it more likely to develop this condition:

• Ethnicity: IgA is more common in people with Asian or white European ancestry
• Celiac disease: An autoimmune disorder that occurs after eating gluten
• Gender: This chronic kidney disease affects men more often than women
• Age: It frequently occurs in males from their teens to late thirties

Family history: In over 10% of cases, IgA nephropathy runs in families, meaning it can be inherited. People who inherit the gene may not show symptoms but could still pass it on

How to Diagnose IgA Nephropathy

Diagnosis helps rule out other conditions like IgA vasculitis, which also cause symptoms like blood in the urine. There are several methods to test for IgA nephropathy:

• Urinalysis: The test helps to detect the presence of protein or blood in the urine
• Urine Protein-Creatinine Ratio (uPCR): This urine test checks the levels of protein and creatinine (a waste product) in the urine
• Kidney biopsy: Involves removing one or several small kidney samples for microscopic observation

Estimated Glomerular Filtration Rate (eGFR): Blood tests assess how well the kidneys can remove waste from the blood

Treatment Options

Currently, there is no cure for IgA nephropathy, and it can be challenging to predict how the condition will affect kidney health in the long term.

Some people only need regular medical tests to monitor the progression of the disease, while others may require medication to control symptoms and prevent the condition from worsening.

Medicine for Treating IgA Nephropathy

Various types of medicines can be used to treat IgA nephropathy. These include:

Immune System-Calming Medication

Also known as immunosuppressants, these strong medicines can lower the body’s defenses. They work by preventing the immune system from producing the proteins that affect the glomeruli. 

However, these medicines can have potential side effects, including high blood sugar, high blood pressure, and a higher risk of infections. It’s important to work with a healthcare provider to choose the right immunosuppressant.

High Blood Pressure Medication

Medications like angiotensin receptor blockers (ARBs) and angiotensin-converting enzyme (ACE) inhibitors are commonly used to lower blood pressure. By doing so, they also help reduce protein loss in the urine.

Omega-3 Fatty Acids

Omega-3 fatty acids, common in fish oil supplements, can help reduce inflammation in the glomeruli with fewer side effects. It’s best to seek a doctor’s guidance when choosing prescription supplements.

Diuretics

Diuretics are medicines that help kidneys remove more salt and water from the body through urine. They can also help control edema- swelling in the feet or hands due to fluid buildup.

Cholesterol-Lowering Medication

People with kidney damage are vulnerable to a heart attack or stroke. Controlling cholesterol with medicines, such as statins, can lower this risk.

Dialysis

When there’s kidney failure, it means 85 to 90 percent of kidney function is lost, and dialysis becomes necessary to remove fluids and wastes from the body.

Dialysis helps manage blood pressure by filtering fluids and wastes from the blood. It can be done using a machine (hemodialysis) or a cleansing solution around the belly lining (peritoneal dialysis).

Kidney Transplant

This treatment is a long-term approach to kidney failure and can save patients from needing dialysis multiple times a week. Studies show that people who opt for a kidney transplant live longer than those who use dialysis.

Discussing this option with a doctor is important to determine the best action.

The Progression of IgA Nephropathy and its Impact on Kidney Function and Overall Health

IgA nephropathy progresses differently for each person, depending on overall health and how well the symptoms are managed.

Some people don’t show symptoms and might not even know they have the disease. Others may have symptoms like blood in the urine but still have properly functioning kidneys. Kidney failure can sometimes occur after a few months or several years.

Effective medication can put the disease into remission, meaning the symptoms no longer appear. However, once the kidney is damaged, about one in four people with IgA nephropathy may experience kidney failure. Others might go into remission or continue to have blood or protein in their urine without serious kidney damage.

How to Manage IgA Nephropathy

If diagnosed with IgA nephropathy, the following strategies can help control its symptoms:

• Lower blood pressure: Limit fat and sodium intake, work out often to enhance weight loss, and take prescribed medications.
• Reduce Protein Intake: Eating less protein can help decrease protein buildup in the kidneys and its appearance in urine.
• Monitor blood pressure at home: Record your blood pressure readings and bring them to your next checkup.
• Eat plant-based foods: Incorporate beans, vegetables, fruits, and whole grains into your diet, as they are fiber-rich.
• Self-education: Use relevant IgAN resources, such as guides, podcasts, apps, and recipes, to learn more about managing the symptoms of IgA nephropathy.

Living with IgA nephropathy can be challenging, especially after kidney failure. Fortunately, various treatment options and lifestyle adjustments can help manage symptoms. Additionally, medical research is ongoing to discover more effective treatments for those with the disease.

One promising development is sparsentan, an FDA-approved drug that doesn’t suppress the immune system. More clinical trials are underway, with organizations like Science 37 leading the research. These trials aim to find new treatment options to reduce IgA buildup in the kidneys and alleviate associated symptoms.