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 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
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
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
Cancer
Cancer: Breast
Cancer: Women's Cancer
The purpose of this study is to find out if the study medicine, XEN1101, can safely reduce seizures.
Study Topics
Epilepsy
Neurology Disorders
