Atrial fibrillation (AFib) is a common cardiac arrhythmia. It happens when your heart rate becomes irregular, often causing a fluttering or racing feeling in your chest. AFib may lead to a higher risk of stroke, heart failure, and other serious problems. In the past, many AFib treatments followed a “one size fits all approach.” Now, doctors are exploring precision medicine to offer more personalized solutions.

What Is Precision Medicine?

Precision medicine focuses on you as an individual. It looks at your genes, lifestyle, and overall health to create a treatment plan that fits your unique needs. In AFib, this might mean analyzing your genetic variants, measuring your ejection fraction (how well your heart pumps blood), and closely monitoring your heart rate to find the best therapy.

How Does It Apply to AFib?

Researchers are learning that AFib may not be the same for everyone. It can appear alongside atrial flutter, acute coronary syndrome, or other heart problems. Some people have persistent and long standing AFib, while others only get short bouts. Your genes may affect how you respond to an antiarrhythmic drug or whether you might need radiofrequency ablation (a procedure that burns or freezes certain heart tissues to restore normal rhythm).

Genetic Profiling

Recent clinical trials often examine genetic variants to see who might respond best to certain treatments. This is important because each person’s AFib may have different triggers. If doctors know the genetic cause, they can focus on therapies that target that cause. This can help reduce complications like cardiac arrest or long-term heart failure.

Personalized strategies are also reshaping how you can join AFib studies. Research programs increasingly invite participants to share genetic data so experts can match you with treatments suited to your exact heart condition. This approach paves the way for better outcomes, as each therapy choice is backed by a thorough look at what makes your case different.

When you take part in these tailored clinical trials, you gain access to promising therapies that go beyond traditional methods. Researchers track your response over time, adjusting your plan if needed. Because the treatment targets the specific genetic variants at play, you may experience fewer side effects and a lower risk of progression to heart failure.

How Close Are We to Curing AFib?

Currently, there is no complete cure for atrial fibrillation (AFib), but significant progress is being made. The goal of treatment is to manage your symptoms, restore a normal heart rhythm and rate, and reduce the risk of serious complications like blood clots and stroke.

Radiofrequency ablation, a procedure that targets the areas in your heart causing irregular rhythms, has shown good success rates for many people. Medications, including antiarrhythmic drugs, also play a crucial role in controlling AFib and preventing episodes. These treatments continue to improve, offering better outcomes for patients.

While researchers are still working on a permanent cure, advances in precision medicine and tailored therapies are making it possible to manage AFib more effectively and improve long-term results. The future of AFib care looks promising, with treatments becoming increasingly personalized to meet your specific needs.

Is Precision Medicine the Future of Healthcare?

Many experts believe that precision medicine will shape the future of healthcare. Instead of using general treatments that may or may not work for you, doctors can tailor therapies based on your unique profile. This approach shows promise in heart health and across many other fields, including cancer and diabetes care.

Precision medicine also focuses on preventing AFib. Identifying genetic risk factors and understanding your specific heart health profile allow doctors to recommend lifestyle changes and preventive measures tailored to you. Early diagnosis through genetic screening can catch AFib before it leads to more serious complications, ensuring timely and appropriate treatment.

The Ultimate Goal of Precision Medicine

Precision medicine aims to provide you with the best possible care by using the latest research and technology. It wants to improve your quality of life, lower your chances of serious events, and help you avoid unnecessary treatments. In AFib, this could mean:

• Picking the right antiarrhythmic drug based on your genetic markers.
• Scheduling radiofrequency ablation only if it fits your specific needs.
• Adapting your care plan if your AFib is persistent and long standing.

The Limitations of Precision Medicine

Despite the promise of precision medicine, there are still challenges:

1. Cost and access: Not all patients have easy access to genetic testing or specialized clinics.
2. Complex data: Analyzing large amounts of genetic and clinical information requires advanced tools and skilled experts.
3. Ongoing research: New methods and medications take time to test in clinical trials, and results must be confirmed before they become widely available.

These limitations do not stop progress, but they do slow it down. Researchers, doctors, and organizations like Science 37 keep working to overcome these barriers.

The Future of Tailored Clinical Trials

Clinical trials are crucial for discovering better AFib treatments. Today, more trials focus on your specific needs. For example, if genetic testing shows that you respond well to a particular antiarrhythmic drug, you might join a trial that studies that drug’s effectiveness in people with similar genes. Trials might also measure your heart rate remotely so that you can participate from home.

Science 37 leads the way by offering patient-friendly trial designs. Their platform makes it easier for you to enroll and stay involved. This helps collect high-quality data, speeding up the process of finding better treatments.

Clinical trials are essential for moving this research forward, and organizations like Science 37 are making these studies more accessible than ever. Our virtual platform allows you to participate from home, reducing travel and streamlining the trial process. This patient-centered model helps us gather valuable data and develop more personalized therapies for AFib, atrial flutter, and other heart conditions.

With precision medicine, you can look forward to treatments designed around your heart’s needs, not someone else’s. The approach aims to improve your day-to-day life and boost your health outcomes over the persistent and long standing.

Asthma research is evolving rapidly, thanks to the rise of digital technology. Clinical trials, once limited by geography and in-person visits, are now breaking barriers through telemedicine, wearable devices, and virtual platforms. These advancements make it easier for you to participate in research studies from the comfort of your home.

This is a game-changer for people living with asthma—a chronic lung disease that causes symptoms like wheezing and shortness of breath. You can contribute to the discovery of new treatments while avoiding the time and stress of frequent clinic visits. At the same time, researchers gain access to real-time data, which speeds up the development of better therapies for improving quality of life.

In this article, we’ll explore how technology is transforming asthma clinical trials and how you can help shape the future of asthma care.

Telemedicine in Asthma Clinical Trials

Telemedicine is one of the biggest technological advancements in health care. It lets you meet with doctors online and avoid frequent in-person visits. This shift is essential for asthma clinical trials, which often require regular check-ins.

Before telemedicine, you needed to travel long distances for each visit. Now, you can participate in a research study from the comfort of your home. This flexibility reduces stress on your schedule, saves you money on travel, and helps you focus on managing your asthma symptoms.

Telemedicine also helps researchers gather valuable data in real-time. During video calls, you can describe any changes in your breathing or quality of life directly to the research team. This feedback helps them detect early signs of asthma exacerbations and adjust your asthma medications if needed. Telemedicine became even more essential during the COVID-19 pandemic because it minimized the need for face-to-face contact, protecting you and the medical staff from potential exposure.

Wearable Devices and Real-Time Data

Wearable devices—like smartwatches, fitness trackers, or specialized sensors—are another way technology is changing how experts study lung disease. They can track your heart rate, oxygen levels, and even your daily steps. Some devices alert you when you might be at risk of an asthma exacerbation. This immediate insight helps you and the research team respond sooner.

Because these devices send data in real-time, your study doctors can see how your body reacts to specific triggers. For example, if your neighborhood has higher pollen levels on a particular day, your device may record an increase in your asthma symptoms. Spotting these patterns allows doctors to recommend adjustments to your treatments, such as using an inhaled corticosteroid more often. You can also receive tips through smartphone apps that connect to your wearable device, helping you stay proactive and comfortable.

Virtual Platforms for Easy Access

Virtual platforms combine telemedicine and wearable device tracking to create a complete online experience. These tools offer everything from secure video appointments to digital instructions on how to use your inhalers. With platforms like Science 37, you can enroll in home studies, schedule online visits, and talk with your research team through chats or video calls.

If you’re worried about missing an in-person visit, rest assured that many studies still include at least one trip to a local clinic or hospital. However, turning most of your research study tasks into digital activities saves time and hassle. Virtual platforms also make it easier for people who live far from major hospitals or clinics to join clinical trials. Improved access to care means more diverse groups of participants, which helps researchers learn how to treat asthma in different populations.

Benefits of Remote Participation

1. Convenience: You can skip frequent trips to the clinic. Most check-ins happen through your smartphone, laptop, or wearable device.
2. Personalized insights: Real-time data lets doctors monitor your progress and provide advice tailored to your needs. If an issue arises, they can quickly suggest changes to your asthma medications.
3. Improved quality of life: Remote participation means fewer interruptions to your daily routine. You spend less time traveling and more time focusing on keeping your asthma symptoms under control.
4. Potential compensation: Some remote research studies are paid. You may receive compensation for your time, effort, and participation.
5. Advancing treatment: Participating in a clinical trial helps researchers test new approaches and medications to reduce asthma exacerbations and improve lung function for future patients.

Overcoming Challenges and Fears

It’s natural to worry about learning new technology. You might wonder if you need special training or expensive devices. The good news is that most virtual platforms are designed to be user-friendly. Study coordinators also provide tutorials and support. If you need to handle new devices at home, a simple guide or online demo often helps you get started.

Another concern is whether you’ll lose the personal touch that comes with an in-person visit. In reality, many participants find that virtual appointments offer more frequent communication with the research team. You can reach out whenever you have questions rather than waiting for a scheduled clinic visit.

How to Get Involved

1. Ask your doctor if you’re a good candidate for home studies or virtual clinical trials. They can help you find active research studies in your area or direct you to online resources.
2. Websites like those run by Science 37 or groups like the Allergy & Asthma Network list current trials. You can see the eligibility criteria, learn about the study goals, and sign up if you qualify.
3. Patient advocacy groups and community health centers often share information on clinical trials. They can also connect you with support networks that have first-hand experience with remote asthma research.
4. Once enrolled, you might need to complete a screening to confirm you meet the study’s criteria. This step ensures the clinical trial is safe and appropriate for your condition.

Looking Ahead

Asthma is a complex lung disease, and each person’s journey is unique. Remote clinical trials offer hope for better, faster, and more personalized asthma treatments. You get the convenience of monitoring your asthma symptoms from home while researchers gain real-time data to develop safer and more effective therapies. As these innovative approaches become more common, they can transform how you and others manage asthma, limit asthma exacerbations, and maintain a higher quality of life.

Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are rare diseases affecting the liver and bile ducts. Though each disorder has distinct causes and symptoms, both can lead to scar tissue formation, increasing the risk of liver failure and the eventual need for liver transplantation.

The limited knowledge of their pathogenesis leads to limited therapeutic options. However, the landscape of novel therapies for cholangiopathies is quickly shifting, offering new treatment opportunities for patients and healthcare providers.

Today, medical research is exploring combination therapies for PBC and PSC to protect liver cells, reduce the chance of infections like Clostridioides difficile infection, and slow or even halt disease progression. And with organizations like Science 37 making clinical study participation simpler, more patients can participate in studies that could improve quality of life and offer hope for better treatments.

Let’s look at the latest advancements and see why combination therapies could shape a brighter future for those affected by these challenging conditions.

What Is Combination Therapy?

Combination therapy means using more than one treatment at the same time to tackle a disease. In the case of PBC and PSC, it often involves medications that reduce inflammation, support bile flow, and protect liver cells from permanent damage. Because each of these liver diseases can cause scar tissue in the bile ducts, doctors hope that working on several fronts will produce better results than relying on a single treatment alone.

Examples of Combination Therapies

Combination therapies for PBC and PSC focus on using multiple treatments to target different aspects of these liver diseases. For example, ursodeoxycholic acid (UDCA), the standard therapy for PBC, is often combined with newer drugs like obeticholic acid (OCA) or bezafibrate. These combinations aim to improve bile flow, reduce inflammation, and lower alkaline phosphatase levels—a key marker of liver damage. Studies have shown that these pairings can help patients who don’t fully respond to UDCA alone.

Other approaches include combining anti-inflammatory drugs with antifibrotic agents to reduce liver inflammation and scar tissue formation, or using biologic therapies alongside standard treatments to manage more severe cases. In PSC, antibiotics like vancomycin are also being studied in combination with anti-inflammatory drugs to address bacterial imbalances and reduce symptoms. These therapies hold promise for slowing disease progression, protecting liver cells, and improving overall quality of life for patients.

The Growing Need for Combination Therapies

PBC and PSC are part of a broader spectrum of chronic liver conditions that include nonalcoholic fatty liver disease (NAFLD), liver cancer, and others. In PBC, the immune system progressively damages the small bile ducts, while PSC often involves a more complex network of inflammation that may also affect the large bile ducts.

Standard therapies, such as bile acid supplements and immunosuppressive drugs, have shown some success in slowing these conditions. However, many patients still risk permanent damage to their liver cells, requiring additional treatments or, eventually, liver transplantation.

Researchers have discovered that combining two or more therapies may not only slow disease progression but also address complications like clostridioides difficile infection that can occur in advanced stages. This dual or triple approach aims to reduce inflammation, enhance bile flow, and protect liver cells from ongoing injury.

How Clinical Trials Drive Innovation

Groundbreaking medical research thrives on well-designed clinical study protocols, thorough blood tests, and advanced imaging tools like a CT scan. In studying PBC and PSC, investigators often track changes in alkaline phosphatase levels—an enzyme that can indicate ongoing inflammation or damage to the liver. These meticulous measurements help researchers determine how effective each therapy is, and identify the combination that offers the most benefit with the fewest side effects.

The past decade has witnessed an expanded focus on combination therapies, driven by collaborations among pharmaceutical companies, academic centers, and patient advocacy groups. Insights gleaned from each clinical study can be rapidly shared and analyzed, allowing researchers to refine their approaches and accelerate drug development. This teamwork is critical: each discovery propels the field forward, offering renewed optimism that severe complications and liver failure might be avoided.

How Science 37 Expands Access to Breakthrough Trials

Accessing rare disease trials has traditionally been challenging due to geographic barriers and the unique expertise needed to treat conditions like PBC and PSC. That’s where innovators like Science 37 step in. The organization harnesses digital platforms, telemedicine, and a network of specialized healthcare professionals to help patients overcome many of the common hurdles to participation—such as traveling long distances or coordinating multiple onsite visits.

How to Join Science 37 Clinical Studies

Joining a Science 37 clinical study is simple and convenient, especially if you’re managing a condition like PBC or PSC. Science 37 lets you participate in groundbreaking research from your place of choice—no need to travel to a doctor’s office or clinic. Your involvement is crucial in advancing treatments for rare diseases like these.

Step 1: Sign Up and Share Your Information

The first step is signing up to join the Science 37 community. You’ll be asked for basic contact information and a brief medical history overview. This helps the team determine if there’s a current clinical study that’s a good fit for you. If not, they’ll stay in touch and let you know as soon as a matching study becomes available.

Step 2: Pre-Qualification and Screening

If a study matches your profile, you’ll move on to the pre-qualification stage. Here, you’ll answer more specific questions about your health through an online screener or by speaking with a team member over the phone. This step ensures that the study is right for you and that you meet its eligibility criteria.

Looking Ahead

Advancements in combination therapies for PBC and PSC mark a significant step forward in the fight against liver diseases that once felt unbeatable. These therapies tackle multiple aspects of the disease at the same time, aiming to prevent permanent damage and lessen the need for liver transplantation.

Organizations like Science 37 are improving trial designs and offering remote participation options, making crucial research accessible to more patients. This means effective treatments could reach those who need them faster.

There is still much work to be done, but the progress in medical research and the teamwork of experts worldwide bring hope to many. For anyone dealing with conditions that might lead to liver failure or other serious health issues, breakthroughs in combination therapies—and easier access to clinical studies—could protect the liver, keep vital liver cells functioning, and ultimately enhance quality of life.

About 795,000 people in the US suffer stroke every year. According to new reports, someone gets a stroke every 40 seconds, and every 4 minutes, someone dies from a stroke. It’s heartbreaking to think about, especially knowing that strokes don’t just take lives—they can also lead to long-term severe disabilities.

Of the 7 million stroke survivors, two-thirds are disabled, and 25% will have another stroke within just five years. But the good news is up to 80% of strokes may be preventable with exercise, better nutrition, and identification of risk factors.

Strokes occur when the brain’s blood supply is interrupted, either by a blockage or a rupture in a blood vessel. Atrial fibrillation (AFib) is a considerable risk factor for stroke. When you have AFib, the upper chambers of the heart beat irregularly and out of sync with the lower chambers. Irregular heartbeats (heart arrhythmia) can cause blood to pool and form clots in your heart. If a clot escapes and travels to your brain, it can block blood flow and cause a stroke.

Innovative Stroke Prevention Strategies in AFib

Currently, there’s no known cure for AFib. But there’s treatment to help control symptoms and prevent complications. Researchers are continuously exploring new ways to treat atrial fibrillation and prevent stroke. Let’s explore some innovative stroke prevention strategies.

Novel Anticoagulants

Novel anticoagulants, also called direct oral anticoagulants (DOACs), are a newer and often preferred option for many patients. These medications target specific clotting factors in your blood to prevent clots from forming.

Types of DOACs

Here are the most common DOACs prescribed for a range of heart diseases, including cardiac arrhythmia:

• Dabigatran: A direct thrombin inhibitor that prevents blood clots by blocking thrombin, an enzyme involved in clot formation.
• Rivaroxaban: A direct factor Xa inhibitor that stops the activity of factor Xa, a protein critical for blood clotting.
• Apixaban: Another direct factor Xa inhibitor known for its effectiveness and lower risk of bleeding.
• Edoxaban: Also a direct factor Xa inhibitor, offering a safe and effective way to reduce stroke risk.

How DOACs Compare to Warfarin

Warfarin has been used for decades. But it's among the top 10 drugs with the highest number of serious side effects reports submitted to the US FDA during the last 20 years. The biggest safety concern of this drug is the risk of major bleeding.

Warfarin also requires frequent blood tests and comes with dietary restrictions because it interacts with foods high in vitamin K. DOACs, on the other hand, offer similar anticoagulation benefits but with fewer complications.

Are DOACs Right for You?

DOACs are often recommended for AFib patients who need anticoagulants, especially if you’re at risk of stroke but don’t want the hassle of constant monitoring. However, they may not be suitable if you have certain conditions, like severe kidney disease.

If you’re currently on warfarin or haven’t started anticoagulation therapy, talk to your doctor about whether a DOAC could be a better option for you. These medications are helping countless people with AFib live safer, more manageable lives.

Left Atrial Appendage Closure Devices

For some patients with atrial fibrillation (AFib), long-term use of blood thinners isn’t an ideal option due to risks like excessive bleeding or other medical conditions. In these cases, left atrial appendage (LAA) closure devices offer an alternative way to reduce the risk of stroke. These devices block the LAA, a small pouch in the upper chamber of the heart where blood clots often form in AFib patients, preventing clots from getting into the bloodstream and causing a stroke.

How LAA Closure Devices Work

These devices are implanted in the heart to seal off the LAA and stop blood from pooling and clotting in this area. Several LAA closure devices are currently available, each with unique features:

• WATCHMAN: This parachute-shaped device is inserted through a catheter and permanently implanted in the LAA. It’s one of the most common and well-studied devices for stroke prevention.
• Lariat: Unlike other devices, the Lariat works by tying off the LAA from the outside of the heart using a pre-tied loop of suture.
• AMULET: A newer device with a plug and a double closure mechanism for added safety and effectiveness.

The Procedure

Implanting an LAA closure device is a minimally invasive procedure. Here’s what you can expect:

1. Preparation: You’ll be given general anesthesia to ensure comfort.
2. Access: The groin area is numbed, and a catheter is threaded through a blood vessel to the upper chamber of the heart.
3. Placement: The device is carefully implanted in the LAA to seal it off.
4. Recovery: You’ll stay in the hospital overnight for monitoring before returning home.

The Role of Clinical Trials in AFib Treatment

Innovative treatments like the ablation procedure, LAA, and DOACs have revolutionized stroke prevention for people with AFib. They significantly lower stroke risk and improve quality of life, but they don’t cure AFib. That’s why clinical trials are still essential.

AFib is a complex condition with persistent and long-standing impacts on heart rhythm and overall health. Current treatments aim to control symptoms, reduce the risk of stroke, and improve daily life, but a true cure remains elusive. Clinical trials focus on addressing these gaps by:

• Testing new formulations of medications to reduce side effects or make them more effective.
• Developing cutting-edge devices and minimally invasive procedures to manage AFib more efficiently.
• Studying how AFib progresses to identify potential cures or preventive strategies.

Participating in a trial gives you access to new treatments that may not be available yet at your local medical center. It also helps researchers gather essential data to improve care for people living with AFib.

How to Qualify for AFib Trials

If you want to join a clinical trial, talk to your healthcare provider first. They can help you determine if you qualify based on factors like age, medical history, and the type of AFib you have (persistent or long-standing).

Many trials are looking for participants who have specific symptoms, like chest pain or heart failure, or those who are struggling to manage their risk of stroke. When you participate in a clinical trial, you’re not just helping yourself—you’re helping others by advancing medical science. Many participants report feeling empowered by their role in shaping the future of healthcare.If interested, speak with your doctor or contact a medical center conducting AFib trials to learn about your options. You can also enroll to participate in Science 37’s pioneering research for heart and blood disorders.

Gout is a leading cause of chronic inflammation arthritis in the US. According to the National Kidney Foundation, gout prevalence more than doubled between the 1960s and 1990s and is estimated at 8.3 million adults (3.9%). But this doesn’t come as a surprise considering the obesity epidemic, lifestyle changes, and the fact that gout has no cure.

Gout Symptoms

Gout often starts suddenly, with intense pain in one or more affected joints. The metatarsophalangeal joint (the big toe joint) is a common target, but other joints, like wrists, ankles, and knees can also be involved. Symptoms typically include:

• Intense joint pain
• Redness, swelling, and warmth
• Extreme tenderness that makes even the lightest touch unbearable
• Fever
• Limited range of motion

These flare-ups may be mistaken for other conditions like rheumatoid arthritis, so getting an accurate diagnosis is essential. There’s no cure for gout. The available medications are designed to either:

• Reduce pain and swelling associated with gout attacks or
• Prevent gout complications by reducing the amount of uric acid in your blood

Which kind of medication is right for you depends on the severity and frequency of your symptoms, as well as other health issues you may have. For example, if you only have occasional gout attacks, an NSAID or colchicine might be enough to manage your symptoms. But if your gout is frequent or severe, or if you have visible signs of joint damage (like tophi), your doctor might recommend medications to lower uric acid levels and prevent future complications.

The personalized approach ensures the treatment plan targets your gout and supports your overall health. However, even with these options, many people still struggle to control their symptoms or avoid flare-ups entirely. That’s why researchers continue to explore new therapies through clinical trials.

These trials let you try out new and innovative treatments that aren’t available yet to the general public. They also give you a chance to contribute to research that could lead to better therapies for managing gout pain, tophaceous gout, and the complications that come with it.

Why Clinical Trials Are Important

Gout can be stubborn. Even though there are treatments to manage pain and swelling or lower uric acid levels, they don’t work the same for everyone. Some people still deal with regular gout flares, especially in areas like the big toe or other affected joints.

Participating in a clinical trial could give you access to treatments designed to:

• Better control inflammation and reduce pain
• Prevent flare-ups by targeting the root causes of gout
• Manage complications like chronic kidney disease or high blood pressure

For example, the Science 37 Tophaceous Gout Study focuses on severe gout cases where tophi (painful lumps of uric acid crystals) develop around joints. If this sounds like what you’re going through, this research could offer you new hope.

New Treatments in Development

Gout treatment has progressed, but some patients still struggle to achieve their treatment goals. Thankfully, new therapies are being developed to address these challenges.

New Anti-Inflammatory Treatments

The inflammation in gout is caused by tiny urate crystals that trigger a strong immune response. New treatments focus on blocking this process:

• IL-1 inhibitors: Drugs like anakinra and canakinumab block IL-1, a key driver of gout inflammation. These are given as injections and are helpful for severe, hard-to-treat cases.
• Caspase inhibitors: Medications like pralnacasan stop the activation of IL-1 by blocking enzymes that fuel inflammation.
• NLRP3 inhibitors: Drugs such as dapansutrile target a key step in the immune response, reducing joint pain and swelling during gout flares.

Better Urate-Lowering Options

Newer therapies are improving how we lower uric acid levels in the blood:

• XO inhibitors: Drugs like topiroxostat and others have been shown in trials to help reduce uric acid production. They're effective even for people with kidney issues.
• Uricolytics: Pegloticase (a modified enzyme) breaks down uric acid to make it easier for the body to remove.
• New uricosurics: Drugs like lesinurad and dotinurad help the kidneys remove more uric acid. They're handy for people whose bodies don't excrete uric acid well.

These advances are giving patients more options, especially for severe or treatment-resistant gout. Institutions like Science 37 are also conducting studies, such as their Tophaceous Gout Study, to develop treatments for people with severe inflammatory gout.

How Clinical Trials Work

Clinical trials aren’t as intimidating as they might sound. First, there’s a screening period to see if you’re a good fit for the study. If you qualify, you’ll get access to the trial treatment while being closely monitored by doctors and researchers.These studies test medications to find better solutions for people like you who need more effective relief. And the best part? Many trials provide treatments at no cost. Science 37 even pays participants and allows them to participate in the clinical trials from the comfort of their own homes.

Is This the Right Step for You?

If your gout isn’t under control, or if you’re dealing with severe symptoms like tophaceous gout or regular gout flares, a clinical trial could open up new possibilities. A trial is also a way to help advance research that might improve the lives of others with gout in the future.

Don’t Forget the Basics

Even as you explore new treatments, small lifestyle changes can make a big difference. Gout has many risk factors, some which are within your control. For example, being overweight increases stress on your joints and raises uric acid levels, but losing weight reduces that risk. Eating too much red meat or drinking sugary beverages also triggers flare-ups—swapping these for healthier food and drinks can help prevent them.

It’s all about making small, sustainable changes. And if you’re unsure where to start, your primary care provider can help you determine what works best for you. They can also identify other risk factors, like certain medical conditions or medications, and help you create a plan to keep things under control.

Be Part of the Future of Gout Treatment

With innovative therapies and ongoing research, the outlook for managing severe or treatment-resistant gout is brighter than ever. Your participation can make a difference. Progress depends on people like you contributing to this critical research. If you or someone you know is living with inflammatory gout, join us in advancing care and finding better solutions.Take the first step—sign up today.

Asthma is one of the most common respiratory diseases in the US. According to the Asthma and Allergy Foundations of America, about 28 million people in the country have asthma or about 1 in 12 people. Despite its widespread occurrence, there’s currently no cure.

Current Treatment for Asthma

Asthma doesn’t have a cure, but treatments are available that can help you control the symptoms and live a normal, active life. These include:

• Rescue inhalers, like albuterol, are a big help during an asthma attack. These asthma inhalers work fast by relaxing the muscles around your airways so you can breathe more easily within minutes.
• Combination inhalers that pair corticosteroids with long-acting bronchodilators. These are great if your asthma is more complicated to manage because they provide longer-lasting relief throughout the day. 
• Inhaled corticosteroids (ICS), like fluticasone, reduce swelling in your airways to prevent symptoms like coughing and wheezing. If you use them regularly, they can stop flare-ups before they even start. ICS are not the same as anabolic steroids used by some athletes.
• For more severe cases, biologic therapies like dupilumab can help target specific parts of your immune system to reduce inflammation and make it easier to manage your asthma.
• Allergy shots can also be effective for allergens like dust mites that may trigger asthma. They help your body become less sensitive to triggers, so you have fewer asthma attacks.
• Doctors also recommend breathing exercises, physical activity, and other lifestyle changes to help manage this lung disease.

Asthma treatments have come a long way, but managing asthma exacerbations remains a challenge for many people. Clinical trials are helping change that by exploring innovative therapies that target the root causes of asthma and provide better long-term control. Let’s look at how these trials work, the exciting treatments being developed, and what this could mean for people with asthma.

How Clinical Trials Are Advancing Asthma Treatments

Clinical trials are research studies that test new medications, therapies, or approaches to managing diseases. For asthma, they’re opening the door to treatments beyond traditional options like metered dose inhalers and inhaled corticosteroids.

When you participate in a trial, researchers study how a new treatment affects asthma symptoms, quality of life, and overall lung function. They also monitor side effects to ensure the treatment is safe and effective. This process helps uncover better ways to control asthma and reduce asthma exacerbations.

Targeting the Immune System with Innovative Therapies

One exciting area of research focuses on how the immune system triggers asthma symptoms. A recent study revealed that a protein called Piezo1 plays a role in calming overactive immune cells in the lungs. These cells, called ILC2s, are often hyperactivated by allergens like dust mites, leading to airway inflammation.

An experimental drug called Yoda1 has shown promise in activating Piezo1. In studies on mice, Yoda1 reduced airway inflammation and asthma symptoms by preventing the immune cells from overreacting. Researchers have even tested Yoda1 on humanized mice—mice engineered with human immune cells—and found similar results. This research study opens the door to new therapies targeting these immune responses in people with asthma.

Although more work is needed to develop drugs like Yoda1 for humans, these findings represent a huge step forward in understanding how to control asthma at its source.

Exploring Severe Asthma in Clinical Trials

Researchers are also running a series of other clinical trials to uncover better ways of managing asthma. Take Science 37, for instance. The organization is conducting a study called the EXHALE Severe Asthma Trials, which aims to find relief for those with severe asthma symptoms. The purpose of this study is to explore new treatment options that could improve asthma control and quality of life for patients who struggle with frequent asthma exacerbations despite current therapies.

The trials include two phases:

• EXHALE-2 and EXHALE-3, which run for up to 56 weeks,

These trials investigate dexpramipexole tablets, a medication designed to reduce lung inflammation and provide better symptom management for severe asthma cases.

Examples of Successful Past Trials

Clinical trials have already reshaped how asthma is treated today. For example, the development of biologic therapies like dupilumab and omalizumab stemmed from extensive research studies. These treatments target specific molecules in the immune system to reduce inflammation and prevent asthma attacks.

Another breakthrough involved improving the design of metered dose inhalers to deliver asthma medications more effectively, making it easier for patients to use them during asthma attacks.

What Clinical Trials Mean for You

Joining a clinical trial can be a great way to try new treatments before they’re available to everyone. It also helps advance asthma research, which could lead to better treatments for millions of people. Plus, clinical trials are carefully monitored, and you’ll get support throughout the process.

For Science 37 clinical studies, you won’t have to pay for any of the study-related assessments. These could include things like:

• Physical exams
• Checking your blood pressure, heart rate, and weight
• An electrocardiogram (ECG)
• A test to check how well your lungs are working (spirometry)
• Lab tests and urine tests
• A pregnancy test if you're someone who can get pregnant

All doctor visits, tests, and medications related to the study are free. You’ll also be paid for your time and travel. Before you start, you’ll get a document that explains everything about the trial. You’ll have plenty of time to read it and ask questions if necessary. Your study team is there to help and answer any questions you have.

Talk to your doctor first if you’re considering joining a clinical trial. They can help you understand the study and whether it’s the right choice for your asthma.

A Brighter Future for Asthma Patients

If you’re ready to be part of this exciting journey and help shape the future of asthma treatment, consider enrolling in a Science 37 clinical trial. Your participation could bring new relief to those with asthma and open doors to innovative therapies. Talk to your doctor today about how you can get involved and make a difference.

Gout pain is a sharp, intense pain that seems to appear out of nowhere. It mostly affects the big toe, though it can also occur in ankles, knees, hands, wrists, hands, fingers, or elbows. The pain is often so bad that it wakes you up at night.

If you’ve dealt with gout pain before, you know how hard it can be to manage. The usual medications may help, but they don’t always provide the relief you’re hoping for or last long enough to prevent the next attack. It’s a frustrating cycle; sometimes, it feels like there is no end in sight.

But, new clinical research is making progress in finding better ways to treat gout. Clinical trials are now looking at options that go beyond the usual medications. Scientists are trying new treatments that target the causes of gout and biologics that work with the immune system.

A Closer Look at Gout

Gout is a type of arthritis that develops when there’s a high level of uric acid in the blood. Usually, uric acid is something your body gets rid of, but when there’s too much of it (hyperuricemia), it can start to form tiny, sharp crystals. These crystals settle into your joints, causing that sudden, intense pain.

When these crystals gather in a joint, it triggers an inflammatory response—meaning your body reacts to the crystals like they’re an injury or infection, bringing on redness, warmth, swelling, and that unmistakable sharp pain. What makes it even trickier is that these flare-ups can come and go. You might have weeks or even months without symptoms, and then suddenly, the pain comes back with a vengeance.

The National Institutes of Health notes gout is the most common type of inflammatory arthritis in adults. It affects about 8.3 million people in the US alone.

Exploring New Treatments for Gout

Traditional gout treatments are designed to lower pain and minimize uric acid levels, but they don’t always work for everyone, especially for those with frequent or severe flare-ups. That’s why researchers are looking at innovative treatment approaches. New medicines in development aim to address the root cause of the condition - like high uric acid levels and inflammation.

Targeted Therapies

One promising approach in hyperuricemia research involves targeted therapies. These treatments focus specifically on the mechanisms that lead to gout flare. For instance, researchers are looking into treatments that can prevent the formation of uric acid crystals, which cause the sharp, intense pain you feel during a gout attack. These treatments aim to block crystal formation at the source to prevent flare-ups before they even begin.

There’s also research into drugs that block specific proteins responsible for inflammation in gout. Unlike traditional pain relievers that manage symptoms, these drugs aim to stop the inflammatory process triggered by uric acid crystals. This approach offers potential for patients who have persistent inflammation despite taking standard medications, bringing a new level of control to those with difficult-to-treat gout cases.

Biologic Treatments

Another area of exploration in clinical trials is biologic treatments. Biologics are made from living cells and work with the immune system. They’re designed to “teach” your immune system to handle the buildup of uric acid better, helping reduce both the frequency and severity of gout flares. This is a fascinating option for people with recurring gout attacks who haven’t had success with other treatments.

These biologics target the specific immune responses that lead to inflammation. They aim to reduce symptoms long-term, potentially giving people greater freedom from gout pain over time.

Addressing Treatment Gaps with Clinical Trials

Clinical trials also address gaps in current treatment of gout options. Many traditional medications can be hard on the kidneys, which is a concern for gout patients who may also be at risk for kidney disease. New treatments are being developed with this in mind, providing safer options for those with kidney-related complications. These trials prioritize minimizing strain on the kidneys and other organs, a critical advancement for those needing treatments that protect overall health while managing gout symptoms.

What to Expect as a Clinical Trial Participant

If you’re considering joining a clinical study for gout treatment, it’s helpful to know what to expect. Clinical trials are organized research studies that evaluate new treatment options under the supervision of medical professionals. Before enrolling, you’ll go through a screening process to determine if the trial is a good match for your specific needs and health conditions. Unlike standard medical visits, health insurance is not required for many clinical trials, as the study typically covers treatment costs.

During the trial, you’ll be closely monitored, which means you’ll have regular check-ins and follow-up appointments to track your progress and evaluate how well the treatment works. This level of monitoring gives you the chance to benefit from new therapies while receiving specialized care. Participants may receive either the experimental treatment or a placebo, depending on the study’s design, but regardless, you’ll be contributing to research that could bring lasting change to gout treatments.

Why Clinical Trials Matter for the Future of Gout Care

Clinical trials help advance gout treatments beyond temporary pain relief. Through these studies, researchers can gather valuable insights into how well new therapies work, what side effects might occur, and how safe they are for different types of patients. These findings are then reviewed by institutions like the Food and Drug Administration (FDA) before new treatments are made widely available. For those with chronic or challenging gout, these research studies offer a glimpse of hope for a better quality of life in the future.

New Possibilities for Gout Patients

As these innovative treatments continue to be tested, the future of gout care looks brighter than ever. The clinical research underway has the potential to reduce the intensity of gout flares and prevent them from recurring altogether, bringing genuine hope to those struggling with challenging cases.If you’re curious about joining a clinical trial, it’s worth exploring the options available through Science 37. We’re seeking volunteers with Tophaceous Gout to participate in a paid, interventional research study. Signing up for a Science 37 clinical trial allows you to be part of something meaningful, making long-lasting treatments accessible to others facing the same battle.

If you live with asthma, you may still have trouble with symptoms even on your regular medication. It’s frustrating, especially when it seems like nothing fully keeps those flare-ups at bay. But you’re not alone in wanting better ways to manage asthma and find lasting relief. That’s why clinical trials are crucial.

Clinical trials often explore new potential treatments and therapies for asthma. They’re the gold standard for testing how well new medications or approaches work, giving doctors and researchers clear insights into what might genuinely help. So, why should you consider joining an asthma clinical trial? This blog will highlight the benefits of clinical trials, what you can expect, and how they might open doors to new solutions designed to help you breathe better.

Asthma Overview

Asthma is a chronic lung disease that affects both children and adults. It’s characterized by inflammation and muscle tightening around the airways, which makes it harder to breathe. According to the Asthma and Allergy Foundation of America, asthma affects around 28 million people in the US, which translates to about 1 in 12 people. Asthma symptoms vary from one person to another, and may include:

• A persistent cough, especially at night
• Chest tightness, making it hard to breathe deeply
• Trouble breathing (shortness of breath), sometimes even when resting
• Wheezing when exhaling – and sometimes when inhaling

These symptoms may worsen when you have a cold or when there’s a change in the weather. Other triggers include smoke, pollen, dust, strong perfume and soaps, animal feathers and furs, etc.

Although asthma has no cure, it can be managed effectively with bronchodilators and steroid inhalers, which help ease symptoms and prevent exacerbations. However, despite advances in asthma management, acute exacerbations continue to occur, posing significant challenges for patients of all ages and placing considerable strain on healthcare resources, and the lives of those affected.

The Role of Asthma Clinical Trials and Why You Should Join

Asthma clinical trials always look for new ways to treat asthma, going beyond what’s already out there. When you join one, you can access innovative treatments before they’re available to the public. You also get to work with specialists who can help you understand asthma on a deeper level and improve your quality of life.

How Clinical Trials Expand Asthma Treatment Options

Clinical trials are constantly testing new asthma medications and therapies to help control asthma better. Some trials work on refining inhaled corticosteroids (ICS), while others look into new treatments like biologics, which target specific cells or proteins involved in asthma. These trials aim to create options to make breathing easier, reduce asthma attacks, and let you enjoy life without worrying about flare-ups.

Why Consider Joining an Asthma Clinical Trial?

There are some great reasons to consider joining a clinical trial if you have asthma:

1. Access to new treatments: You’ll be among the first to try new asthma medications or therapies. If your current treatment isn’t fully working, these new options could be worth exploring.
2. Help shape future asthma care: You contribute to necessary research that can improve asthma care. Your experience helps doctors understand what treatments work best for different people.

Personalized attention and care: Clinical trials give you access to specialists who will check on you regularly. They’ll consider your medical history, asthma type and needs to provide you with better insights into managing your asthma.

What to Expect with Safety in Clinical Trials

Safety is always the priority in clinical trials. Before you begin, the trial team will review any risk factors and help you understand the process. You’ll have regular check-ins with the medical team at the research center to monitor your health and track how well the treatment works. They’re there to support you every step of the way, keeping you informed and safe.

The Benefits of Clinical Trial Involvement

Here are some reason why you should participate in a clinical trial:

1. Many trials focus on treatments that aim to reduce asthma symptoms long-term, helping you go about your day without as many disruptions.
2. In a clinical trial, you’re monitored closely, often for a set time, like 12 weeks. Some, like Science 37’s EXHALE-2 and EXHALE 3, may run for up to 14 months. This regular check-in helps doctors see how your body responds and can make a big difference in how well the treatment works.
3. When you join the trials, you become a part of valuable asthma research that could change how asthma is treated. This experience not only brings you closer to experts but also offers a glimpse into the latest in clinical research for pulmonary disease.
4. Many clinical trials cover the costs associated with participation, including medication, visits, and tests. This can ease the financial burden of managing asthma and accessing cutting-edge treatments.

How Clinical Trials Shape the Future of Asthma Treatment

Each clinical trial adds to what doctors and researchers know about asthma. Even if a trial doesn’t lead to a new treatment, it provides insights that can improve future medical trials and help others with asthma down the line. Your involvement makes a lasting impact and brings us all closer to better, more personalized asthma care.

Ready to Explore an Asthma Clinical Trial?

Deciding to join a clinical trial is a big step, but it’s one that offers hope for you and countless others with asthma. If you’re interested, contact a local research center to find out about ongoing studies and see if one is a fit for you. You can also join Science 37’s Severe Asthma Study.Science 37's study gives you a chance to try Dexpramipexol tablets that could help with asthma. All related assessments will be at no cost to you and may include vital signs checks, physical examinations, pregnancy tests, clinical lab tests and urinalysis, lung function tests, and ECG. You will also be compensated for your time and travel for visits.

The bile ducts in the liver are lined by special cells called cholangiocytes. These cells help produce, adjust, and move bile—a digestive fluid—from the liver to the intestines. When cholangiocytes don’t work correctly, it can cause long-term bile duct diseases called cholangiopathies, which are severe and can become disabling.

Two major types of these bile duct diseases are Primary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC). Both are thought to be caused by the immune system mistakenly attacking bile ducts, although the exact causes are still unknown. Here’s a simple breakdown:

Primary Biliary Cholangitis

• Affects tiny bile ducts inside the liver.
• Slowly damages and scars these small ducts.
• Once called "primary biliary cirrhosis," the name changed to reduce stigma and reflect its true nature.
• PBC is rare, affecting 1.91 to 40.2 people per 100,000.

Primary Sclerosing Cholangitis

• Causes scarring in medium to large bile ducts inside and outside the liver.
• Often leads to liver failure or cancer in the liver and bile ducts.
• PSC is also rare, with 1 to 16 cases per 100,000 people.

Both these autoimmune liver diseases involve inflammation and scarring, which can eventually cause liver failure. Right now, there’s no cure for PBC or PSC. Treatments exist but mainly aim to slow disease progression and manage symptoms.

For PBC, medications can sometimes help, but they don’t work for everyone. With PSC, the options are even more limited. Most patients are only able to manage symptoms and hope their disease doesn’t worsen too quickly. In severe cases, patients often need a liver transplant. This is why clinical trials are essential for people with PBC and PSC.

Why Clinical Trials Are Important for PBC and PSC

Clinical studies test the efficacy and safety of new therapies that could improve liver health, reduce symptoms, and potentially halt or even reverse some of the liver damage caused by these diseases. Such trials bring real hope that people with PBC and PSC might have access to treatments that could change the course of these conditions. The latest PSC research aims to:

• Slow down disease progression: Trials test new treatments to see if they can reduce inflammation and prevent scarring (known as liver fibrosis), which could slow down the worsening of PBC and PSC.
• Improve quality of life: Clinical trials often study treatments that target symptoms like fatigue and itching, which can be deeply disruptive to daily life.
• Explore new drug options: Some trials focus on investigational drugs that might adjust the immune response or target specific markers like alkaline phosphate levels linked to bile duct inflammation.

How Clinical Trials Are Structured

Clinical trials progresses through phases to ensure new treatments are safe and effective. Knowing these phases can help you understand the process and what to expect if you decide to participate:

Phase 1: The first step tests whether a new drug is safe, often with a small group of people. Researchers look for any side effects and try to find the proper dosage.

Phase 2: This phase expands to test how well the treatment works, still monitoring safety but with a larger group of participants.

Phase 3: Now, the treatment is tested on a much larger scale, often compared to standard treatments. This phase confirms its effectiveness and checks for any rare side effects.

Phase 4: After approval, these studies continue to monitor the long-term safety and effectiveness of the treatment.

Each phase helps researchers confirm that a treatment is both safe and effective before it becomes available to the broader public.

Who Can Participate in a Clinical Trial?

Eligibility criteria for clinical trials vary depending on the study. Researchers typically look for specific characteristics to ensure the trial results are accurate and meaningful. You may be eligible if you:

• Have been diagnosed with PBC or PSC
• Experience particular symptoms or disease stages that align with the trial’s focus
• Meet health criteria, which could involve age, lifestyle factors, or medical history

Your doctor can help you determine whether a specific trial might be a good fit and whether the potential benefits align with your health needs.

The Benefits of Joining a Clinical Trial

For many people, clinical trials can offer a unique opportunity to explore treatments that aren’t yet available to the public. Here are a few reasons why you might consider participating:

• Access to new treatments: Clinical trials often give you access to investigational drugs that may be more effective than current treatments, mainly if you’ve found limited success with existing options.
• Close medical monitoring: During a trial, medical professionals closely monitor your body’s response to the treatment, which can help detect any issues early on.
• Contributing to future breakthroughs: Participating means helping to advance research for PBC and PSC. Every trial brings researchers closer to understanding these diseases, potentially leading to more treatment options for everyone in the future.

Important Considerations Before Joining

While clinical trials offer many benefits, it’s essential to consider all aspects before deciding to join. Here are some important points to keep in mind:

• Understanding the risks and benefits: Trials may involve investigational drugs that aren’t fully understood, so it’s crucial to weigh the potential benefits against possible side effects. Researchers will explain these risks in detail during the informed consent process.
• Commitment and time requirements: Some trials require frequent visits, ongoing tests, and regular monitoring. Ensure you understand what’s involved to see if it’s feasible and can fit within your lifestyle.
• Potential for a placebo: In some trials, especially in the early stages, you might receive a placebo (an inactive substance) instead of the experimental drug. This is often done to ensure accurate results. However, you won’t know in advance if you’ll receive the actual treatment or placebo.

Finding a Clinical Trial for PBC or PSC

If you’re interested in participating, talk to your doctor about clinical trials in your area, particularly those focused on PBC or PSC. The United States has several public health databases where you can find trials, including the National Institutes of Health (NIH) and clinical trial listings on hospital and research center websites. You can also go directly and sign up for Science 37 clinical trials. Science 37 offers decentralized clinical trials that allow you to participate from the comfort of your home.

Clinical trials are pushing the boundaries of what’s possible for liver diseases like PBC and PSC. With new studies focusing on slowing liver fibrosis, managing symptoms, and exploring novel immune responses, the current research will likely shape tomorrow’s treatment options. Some trials even aim to address complications like liver cancer, making them critical for long-term liver health.

Tuberous Sclerosis complex (TSC) is a rare genetic disorder that causes benign tumors to grow in various parts of the body. These tumors are benign or noncancerous, but they can still cause significant complications depending on where they form. For example, tumors in the brain can lead to seizures or developmental delays, while those in the kidneys, lungs, or heart can impair normal function.

TSC is often first found during infancy or childhood. But in some cases, it can have such mild symptoms that it isn’t diagnosed until adulthood or is never diagnosed at all. People with less severe cases may have only a few effects and a normal lifespan.

Currently, there’s no cure for TSC, and there’s no way to predict the course of the disorder and how bad it will get. However, treatments are available to manage the symptoms. Researchers are also working hard to develop new therapies that could revolutionize how the condition is treated. While current treatments focus on managing symptoms, researchers are exploring options that target the underlying causes of the disorder.

Overview of Tuberous Sclerosis

TSC is an uncommon disorder that affects less than 50,000 people in the US. It causes clusters of cells to grow and become tumors that form in different parts of the body, including the brain, heart, eyes, kidneys, lungs, skin, and other organs. These tumors are typically benign, meaning they are noncancerous. However, depending on their size and location, they can still lead to significant health issues.

Symptoms and Complications

Mild TSC might not cause complications. But when the tumor is big, or when it grows on organs like the lungs, kidneys or brain, it can lead to complications like:

• Seizures
• Developmental delays
• Infantile spasms
• Impaired kidney function
• Cognitive impairments
• Skin issues
• Dental changes
• Kidney failure
• Renal cell carcinoma
• High blood pressure
• Respiratory issues
• Heart complications
• Lung failure
• Learning disability
• Increased risk of cancerous tumors in the brain and kidneys
• Vision damage
• Status epilepticus

What Causes TSC?

TSC is a genetic condition caused by mutations in TSC1 and TSC2 genes. These genes play an essential role in controlling cell growth and division. Your body has built-in mechanisms, including tumor-suppressing proteins, that ensure cells grow at a normal rate and maintain a healthy size.

When mutations in the TSC1 or TSC2 genes disrupt this regulation, cells may grow too quickly or become larger than they should be. Without the right balance, an excess of these oversized cells can crowd out normal tissue. This uncontrolled growth leads to the formation of benign tumors in various organs.

Risk Factors

Tuberous sclerosis can either result from a random cell division error or a gene change passed down in the family. Experts say that about two-thirds of people with TSC have a new change in either the TSC1 or TSC2 gene, and one-third get a gene change from their family members, specifically parents. If you have TSC, you are 50% likely to pass the changed gene to your biological children. Genetic testing can give valuable information to help assess the risk of passing TSC to future generations and guide family planning decisions.

Treatment for tuberous sclerosis

Tuberous Sclerosis Complex is a lifelong condition without a cure, but several effective treatments help manage symptoms and improve quality of life. In the past, medications like Vigabatrin have been a go-to choice for managing TSC-related infantile spasms and focal seizures in young children. Many doctors prescribe Vigabatrin because it has shown effectiveness in controlling seizures, especially when started early.

Ketogenic Diet

Another option is the ketogenic diet, which is high in fats and low in carbohydrates. This diet has helped reduce seizure frequency in over 90% of patients after six months. However, it's worth noting that more recent studies have shown a decrease in effectiveness over longer follow-ups.

mTOR Inhibitors

Recently, there’s been a shift in focus from simply alleviating symptoms to tackling the root causes of TSC. The discovery of mTOR inhibitors, such as everolimus, marks a significant advancement in TSC treatment. Initially approved for tumor reduction, clinical trials have shown that everolimus can reduce seizure frequency and improve overall health in TSC patients.

Clinical trials have shown that this medication can shrink tumors like subependymal giant cell astrocytomas (SEGAs) in the brain and angiomyolipomas (AMLs) in the kidneys, which are common in people with TSC. One study found that about one-third of participants experienced a notable reduction in tumor size within six months of starting everolimus, with many reporting fewer and less intense seizures.

Vagal Nerve Stimulation

Another innovative approach is vagal nerve stimulation (VNS), which uses electrical pulses to modulate brain activity and decrease seizure frequency. While VNS may not be suitable for everyone, it offers an alternative for those who don’t respond well to traditional medications.

Cannabidiol

Cannabidiol (CBD) has also gained attention as a treatment option for managing seizures associated with TSC. The FDA and EMA approved the use of CBD as a potential adjunctive treatment for patients aged two years and older. Its long-term efficacy was evaluated in the open-label extension (OLE) of the randomized, placebo-controlled phase 3 trial GWPCARE6. During the first 12 weeks of the OLE, participants saw an average monthly seizure frequency decrease of 54% compared to the baseline from the randomized phase. This positive effect persisted for up to 48 weeks, with reductions ranging from 54% to 68%.

 

Surgical Options

Surgery is sometimes used to help control seizures or remove growths. However, surgery isn’t always a complete solution, and some people may still have seizures afterward. That's why doctors must do a thorough test beforehand to see if surgery is the best option based on individual cases.

There are newer, less invasive surgery options, like using heat-guided therapy, that can help reduce seizures in some patients. For certain brain tumors (called SEGAs), surgery is necessary if they are causing symptoms. Still, if the tumor is growing without causing problems, doctors might recommend a combination of surgery and medication (mTOR inhibitors) to manage it.

Surgery addresses symptoms but doesn’t cure the condition. It also carries inherent risks. For example, laser surgery or cryotherapy used in treatments for facial angiofibroma reduces lesions but is often painful and leaves scars. Brain surgery comes with a risk of damaging nerves or blood vessels, which can cause strokes or neurological deficits, some of which may be permanent.

A Promising Future

As our understanding of TSC deepens, so does the potential for innovative treatments. Ongoing research into mTOR inhibitors and other targeted therapies offers hope for individuals living with TSC. While these treatments are not panaceas, they represent a significant shift toward addressing the complexities of the disease rather than simply managing its symptoms.

On top of that, researchers are actively investigating innovative therapies through clinical trials. These studies focus on targeted drug therapies and genetic interventions that aim to address the root causes of TSC. Organizations like Science 37 are helping to lead these efforts, and bring hope fo safe and effective treatments.

These innovative therapies are still being tested, but they could change how we manage TSC and improve the quality of life for many patients.