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Studying the Medicine Apitegromab for Babies with Muscle Weakness
Spinal muscular atrophy is a disease that makes muscles weak and hard to move. The purpose of this study is to test a new medicine called apitegromab. We want to see if it is safe and helpful for babies under 2 years old who still have trouble moving even though they already got another treatment called survival motor neuron therapy.
Study Topics
Genetic Disorders Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles Children, Infants, Teen Health
Comparing Different Migraine Medicines
The purpose of this study is to learn more about three migraine medicines: atogepant, propranolol, and topiramate. Our researchers want to see which one helps prevent migraines the best. Volunteers in the study will try one medicine and keep track of their headaches. The results will help doctors and patients choose the best medicine for migraine prevention.
Study Topics
Learning About Patient Experiences with HyQvia for Nerve Disease
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a long-term illness where the body's defense system attacks the nerves, making people feel weak and numb. This study aims to learn about the experiences of people with this disease who are using a medicine called HyQvia. We want to understand their symptoms, how the disease affects their daily lives, and their treatment experiences. The information we gather may help others in the future by improving our understanding of CIDP and making future research studies better.
Study Topics
Comparing Two Treatments for Myasthenia Gravis
Myasthenia gravis (MG) is a rare disease that makes it hard for nerves and muscles to work together. The goal of this study is to compare two medicines, nipocalimab and efgartigimod, to see how well they work and how safe they are for people with this disease. The study will also look at how safe and helpful nipocalimab is for people who switch from efgartigimod.
Study Topics
Neurology Disorders Rare Disease Arthritis, Lupus, and Autoimmune Disorders
Study to See How Well the Study Drug EPX-100 Works for Treating Dravet Syndrome
The purpose of this study is to see how well the study drug, EPX-100, works for treating Dravet syndrome. We also want to learn more about possible side effects.
Study Topics
Studying New Ways to Reduce Caregiver Stress
The purpose of this study is to see if listening to nature sounds and using a new technology called CR can help people who take care of loved ones feel less stressed. Taking care of someone can be stressful and affect your health. This study will test if CR can help reduce stress symptoms like anxiety, trouble sleeping, and changes in heart rate in a small group of people.
Study Topics
Healthy Volunteers Mental Health Neurology Disorders Sleep Brain and Memory
Care Program for Adults with Epilepsy Dealing with Anxiety and/or Depression
The purpose of this study is to find out if emotional quality of life can be improved for those with epilepsy and anxiety and/or depression. We want to see if regular calls or visits with a care manager can help with the anxiety and/or depression symptoms.
Study Topics
Learning More About Treating Childhood Absence Epilepsy and Juvenile Absence Epilepsy with Brivaracetam
The purpose of this study is to see how well the study drug, brivaracetam, treats childhood absence epilepsy or juvenile absence epilepsy. We also want to learn more about any possible side effects it may cause.
Study Topics
Studying CereGate Therapy in Patients Diagnosed with Parkinson's Disease
The purpose of this study is to learn more about a new therapy called CereGate. We want to see if this type of therapy can reduce freezing movements in patients with Parkinson's Disease.
Study Topics
How the Brain Makes Decisions in Patients with Parkinson's Disease
The purpose of this study is to look at how the brain responds to information when it comes to decision making.
Study Topics
Study to Find Out How Well Inebilizumab Works for Treating Brain Inflammation
The purpose of this study is to find out how well the study drug, Inebilizumab, works for people newly diagnosed with anti-NMDAR encephalitis. Anti-NMDAR is brain inflammation caused by antibodies.
Study Topics
Online Learning Tool to Help People with Migraines
The purpose of this study is to see how well an online learning tool works for people with migraine. We need feedback to help ensure this learning tool is usable for people with migraine.
Study Topics
The HEALEY Trial: Learning More About Different Treatments for ALS
The purpose of this study is to learn more about multiple treatments for amyotrophic lateral sclerosis (ALS). We want to find out if different treatments have an effect on ALS. We also want to find out if these treatments are safe to take without causing too many side effects.
Study Topics
Concussion Symptoms in Young People Ages 11-17
The purpose of this study is to learn more about concussion symptoms in young people ages 11 to 17. This may help us develop future treatments.
Study Topics
Learning More About the Medicine XEN1101 for Treating Seizures
The purpose of this study is to find out if the study medicine, XEN1101, can safely reduce seizures.
Study Topics
BE WELL with Migraine
The purpose of this study is to test a non-drug, virtual treatment designed to help improve the lives of people with migraine.
Study Topics
Learning More About Duchenne Muscular Dystrophy Patients and Their Treatment
The purpose of this study is to get a better understanding of patients with Duchenne muscular dystrophy who were treated with Eteplirsen, Golodirsen, or Casimersen. We want to learn more about how well those medications worked for treating Duchenne muscular dystrophy.
Study Topics
Treating Uncontrolled Seizures with the Study Drug NRTX-1001
The purpose of this study is to learn more about treating uncontrolled seizures with the study drug NRTX-1001. We want to see how the study drug works and find out what side effects may be caused.
Study Topics
Treating Focal Onset Epilepsy with Study Drug BHV-7000
The purpose of this study is to find out how well the study drug, BHV-7000, works for treating focal seizures. We also want to learn more about side effects that may be caused by the study drug.
Study Topics
New Migraine Medicine for Teens
The purpose of this study is to learn more about a new medicine called Rimegepant to help teenagers with chronic migraine. We want to see if this medicine is safe and useful to prevent chronic migraine.
Study Topics