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.
Study Topics
Healthy Volunteers
Neurology Disorders
Brain and Memory
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.
Study Topics
Healthy Volunteers
Rare Disease
Bones, Joints, Muscles
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.
Study Topics
Neurology Disorders
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.
Study Topics
Neurology Disorders
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
Movement Disorder
Neurology Disorders
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.
Study Topics
Genetic Disorders
Men's Health
Rare Disease
Bones, Joints, Muscles
Children, Infants, Teen Health
The purpose of this study is to find out if the study drug, XEN1101, can reduce seizures. We also want to see if it is safe to use.
Study Topics
