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How Myotonic Dystrophy Type 2 Affects the Brain
Myotonic dystrophy affects the muscles and other body systems. The purpose of this study is to look at the impact myotonic dystrophy type 2 has on the brain. We also want to look at brain-based symptoms like problems with thinking, memory or motivation.
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Finding Muscle Changes in Myotonic Dystrophy Patients
Myotonic dystrophy (DM) is a disease that causes muscles to weaken and waste away. The purpose of this study is to find the best ways to measure muscle weakness and changes in patients with DM using MRI scans. Understanding these changes will help plan future studies to track the disease and test new treatments. We are seeking healthy volunteers as well as volunteers who have been diagnosed with myotonic dystrophy.
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Studying the Drug AOC 1001 for Patients with Myotonic Dystrophy Type 1
The purpose of this study is to learn more about the study drug AOC 1001. We want to see how safe and useful the study drug is for the treatment of myotonic dystrophy type 1.
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Studying the Drug VX-670 for Adults with Myotonic Dystrophy Type 1
The purpose of this study is to learn more about the study drug VX-670. We want to find out if it is safe and if adults with myotonic dystrophy type 1 can handle it well.
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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
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.
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Tracking the Long-Term Safety of the Medicine AGAMREE
Duchenne muscular dystrophy (DMD) is a disease that makes muscles get weaker over time. The purpose of this study is to track the long-term safety of a medicine called AGAMREE for this disease. People in the study will have yearly checkups and answer questions about their health. We are looking for volunteers who have DMD and are currently taking AGAMREE.
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Genetic Disorders Men's Health Rare Disease Bones, Joints, Muscles Children, Infants, Teen Health
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.
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