Support for Duchenne
Committed to people and their families living with Duchenne
What is Duchenne?
Duchenne muscular dystrophy is a genetic disorder that causes severe and rapid muscle loss and weakness. People living with Duchenne begin to lose their ability to walk without assistance by their early teens and nearly all will require the use of a wheelchair by the time they are in their mid-teens. Currently, there is no cure for Duchenne but there are approved therapies and clinical trials are ongoing. Preserving muscle function is an important part of managing Duchenne muscular dystrophy.
Watch our
Muscle Town video
Discover how muscles work (and why they sometimes don’t in Becker and Duchenne muscular dystrophy) their role in our bodies, the proteins involved, and more.
Watch our NSAA video
Learn how the NSAA, or North Star Ambulatory Assessment, is used in clinical research and care to measure functional progression in individuals with Duchenne or Becker.
Resources
Parent Project Muscular Dystrophy (PPMD)
Fights every single battle necessary to end Duchenne. The organization also supports people and families with Becker.
Jett Foundation
Dedicated to funding Duchenne research and direct service programming for families impacted by Duchenne and other neuromuscular disorders.
Muscular Dystrophy Association (MDA)
Empowers people living with neuromuscular diseases and looks to accelerate research, advance care, and advocate for the support of families living with muscular dystrophy, ALS, and related neuromuscular diseases.
CureDuchenne
Committed to improving the lives of everyone affected by Duchenne through accelerating research to find the cure, improving care and empowering the Duchenne community.
The Akari Foundation
Educate and empower the Hispanic community about rare diseases, help with resources, awareness, advocacy, and education, entirely in Spanish, specializing in Duchenne muscular dystrophy.
Educar y empoderar a la comunidad hispana sobre enfermedades raras, ayudar con recursos, concientización, defensa y educación, completamente en español, especializándose en distrofia muscular de Duchenne
TREAT-NMD
A global network of experts in the neuromuscular field. Everyone in the network helps the organization work toward the mission to accelerate the development of effective
treatments; and establish the best in diagnosis and care for people worldwide.
About Edgewise
Duchenne Clinical Trials
Edgewise is currently conducting two clinical studies for Duchenne muscular dystrophy called LYNX and FOX. There are advantages and disadvantages to participating in a clinical trial. Talk to your doctor or explore the links below to learn more about whether a trial is right for you.
How do others live with Duchenne?
Meet the Romito Brothers.
Brothers. Explorers. Gamers.
Living with Duchenne.
What is Sevasemten?
Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to preserve and protect unstable muscle against contraction-induced injury in individuals living with rare skeletal muscle disorders, including Becker and Duchenne. Clinical trials for sevasemten are ongoing.
Sevasemten is an investigational therapy that has not been approved for use in Becker muscular dystrophy or Duchenne muscular dystrophy by any regulatory agency, as its safety and effectiveness have not been established for the treatment of these diseases.
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