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 January 2026
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
All India Institute of Medical Sciences ( AIIMS), New Delhi, India
Trinity of Care:Academic and Clinical
Children’s Cardiomyopathy Foundation
CCF's mission is to accelerate the search for causes and cures for pediatric cardiomyopathy through increased research, education, awareness, advocacy, and support to affected children and their families.
Cure Rare Disease
Developing a novel drug development framework to allow for the creation of treatments for rare and ultra-rare genetic neuromuscular conditions.
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.
Family, Friends and Duchenne, Inc.
At Family, Friends and Duchenne, we focus on education, support and social inclusion worldwide for anyone whose life has been touched by an individual with Duchenne.
Girls Chronically Rock
Girls Chronically Rock empowers and inspires people in the disability community through fashion, storytelling, and advocacy. Our mission is to create visibility, spark confidence, and promote inclusion by combining style with purpose.
Heart of a Giant Foundation
The mission of the HGF is to achieve better health outcomes for heart diseases and related chronic health conditions in our communities and enhance the quality of life through health education, care support, and connection to community resources.
HeartCharged
Ending preventable deaths from undiagnosed heart conditions and sudden cardiac arrest, especially in young people, and providing patient-to-patient support.
Help Hope Live
Help Hope Live believes a medical crisis shouldn’t become a financial crisis. Help Hope Live supports community-based fundraising for people with unmet medical expenses and related costs due to cell and organ transplants or catastrophic injuries and illnesses. These efforts play a critical role in helping clients access the care and equipment they need to heal, live, and thrive.
Hunter’s Hope Heroes for Duchenne and DEGS1 Leukodystrophy
Hunter’s Hope exists to reduce the stressors for local families affected by Duchenne Muscular Dystrophy and DEGS 1 Leukodystrophy by eliminating barriers to accessible resources, funding research to find effective treatments and improving community understanding of these rare diseases.
Jett Foundation
Jett Foundation empowers people and families impacted by Duchenne muscular dystrophy through the development of transformative programming, educational opportunities, and ongoing support for every stage of a Duchenne journey.
Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) fights to end Duchenne. We accelerate research, raise our voices to impact policy, demand optimal care for every single family, and strive to ensure access to approved therapies.
Patient Access Network (PAN) Foundation
The Patient Access Network (PAN) Foundation is a national patient assistance organization that helps underinsured people with life-threatening, chronic, and rare diseases pay for their out-of-pocket treatment costs, while advocating for more affordable care.
Syndromes Without A Name (SWAN) Australia
Provide information, support and advocacy to families caring for a child with an undiagnosed or rare genetic condition.
TNPO2 Foundation
Develop personalized therapeutics for ultra rare genetic neurodevelopmental diseases, especially those arising from mutations in the TNPO2 gene.
The Akari Foundation
Our Mission Educate and empower the Hispanic community about rare diseases, help with resources, awareness, advocacy, and education, entirely in Spanish, specializing in Duchenne muscular dystrophy.
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Clinical Trials
For a list of clinical trials in this disease area, please click here.