Synonyms: DMD | Severe dystrophinopathy, Duchenne type
A rare genetic muscular dystrophy characterized by rapidly progressive muscle weakness and wasting due to degeneration of skeletal smooth and cardiac muscle.
Data from Orphanet are used to provide information on a disease's name, synonym(s), and overview. Reference: Access aggregated data from Orphanet at Orphadata. Orphadata: Free access data from Orphanet. © INSERM 1999. Available on http://www.orphadata.org. Data version September 2024
Newly diagnosed with
Duchenne muscular dystrophy?
Our RARE Concierge Services Guides are available to assist you by providing information, resources and connections as you navigate your rare disease journey.
Advocacy Organizations
CureDuchenne
Our mission is to improve the lives of everyone affected by Duchenne muscular dystrophy through accelerating research to find the cure, improving care and empowering our community.
DDX3X Foundation
Our mission is to connect families, resources, and the medical community to advance research for a treatment or cure to DDX3X Syndrome. Our ultimate goal is to accelerate brain function in individuals affected by DDX3X Syndrome through advances in cell and gene therapy and pharmaceuticals.
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Clinical Trials
For a list of clinical trials in this disease area, please click here.