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Using a Small Device to Track Muscle Changes in ALS
The purpose of this study is to find out if a new medicine called omecamtiv mecarbil can help people with serious heart failure. The medicine may help the heart pump better and lower the chance of problems like hospital visits or other serious health issues. We hope this research will help doctors treat heart failure in the future.
Study Topics
Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles
Studying the Medicine CREXONT for Parkinson's Disease
The purpose of this study is to learn more about the medicine CREXONT. We want to see if this medicine is safe and useful for patients with Parkinson's disease in their daily lives.
Study Topics
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
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
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
Creating a Database to Learn More About Vercise Deep Brain Stimulation in Patients with Parkinsons Disease
Parkinson's disease affects nerve cells and causes tremors and muscle stiffness. It can also cause difficulty with speech and walking. The purpose of this study is to find out how brain stimulation effects patients with Parkinson's disease.
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
Studying the Medicine VX-670 for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 is a disease that makes muscles weak and hard to relax, and it can also affect other parts of the body like the heart and stomach. The purpose of this study is to see if a new medicine is safe and works well for people with this disease. Our researchers will see if the medicine can help with muscle problems and check for any side effects over two years.
Study Topics
Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles
Studying a New Medicine for Muscle Weakness Diseases
The purpose of this study is to see if a new medicine called anifrolumab helps people with muscle weakness diseases called polymyositis and dermatomyositis. The medicine will be given as a shot under the skin, along with regular treatment. The study will check if this medicine helps people feel better and need less of their usual medicine.
Study Topics
Movement Disorder Neurology Disorders Arthritis, Lupus, and Autoimmune Disorders