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 April 2026
Newly diagnosed with
Duchenne muscular dystrophy?
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Advocacy Organizations
All India Institute of Medical Sciences ( AIIMS), New Delhi, India
Trinity of Care:Academic and Clinical
Association Aux Pas du Coeur – Côte d’ivoire
Our organization wants to raise awareness and recognition of rare diseases in Ivory Coast. Our mission is to: Raise awareness and campaign to help with the diagnosis and free therapeutic care of patients. Request and/or contribute to actions relating to the training of the medical profession to be able to make a final diagnosis and ensure continuous monitoring of patients easily.
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.
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.
Fundacion Powerchair Football Argentina
To develop Powerchair Football in Argentina in order to integrate people with motor disabilities (SMA, Duchenne muscular dystrophy, Achondroplasia, Rare Diseases) into sports practice and to promote their social inclusion, based on empowerment, independence, and the creation of new bonds and friendships.
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.
HeartCharged
Ending preventable deaths from undiagnosed heart conditions and sudden cardiac arrest, especially in young people, and providing patient-to-patient support.
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.
Muscular Dystrophy Pakistan
Muscular Dystrophy Pakistan is a patient-led nonprofit organization dedicated to raising awareness, promoting early diagnosis, supporting affected families, and advocating for equitable healthcare access for individuals living with muscular dystrophies and other rare genetic diseases across Pakistan, especially in underserved communities.
Neuromuscular Disease Foundation
The Neuromuscular Disease Foundation's (NDF) mission is to enhance the quality of the lives of people living with GNE Myopathy (also known as HIBM) through advocacy, education, outreach, and funding clinical research focused on treatments and a cure.
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.
Parent Project Muscular Dystrophy, India
Solution for sustaining the wellbeing of DMD kids
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.
RareDNA Foundation
Dedicated to improving outcomes for individuals and families affected by rare DNA heart mutations by advancing awareness, access and action. We increase awareness among medical professionals and the public, provide education and resources on diagnosis and treatment, support scientific research, and advocate for faster diagnostic pathways, giving families the knowledge and care they need.
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.