Duchenne muscular dystrophy
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.
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
Jett Foundation
Our mission at Jett Foundation is to extend and enrich the lives of individuals affected by Duchenne muscular dystrophy and other neuromuscular disorders.
The Akari Foundation
Mission: To educate, empower & advocate for the Hispanic Immigrant community, help with Resources, advocacy & education in rare diseases, specializing in Duchenne Muscular Dystrophy.
All India Institute of Medical Sciences ( AIIMS), New Delhi, India
Trinity of Care:Academic and Clinical
Cure Rare Disease
Developing a novel drug development framework to allow for the creation of treatments for rare and ultra-rare genetic neuromuscular conditions.
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.
Family, Friends and Duchenne, Inc.https://familyfriendsandduchenne.org/?amp=1
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.
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.
Cure Duchenne
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.
Girls Chronically Rock
Creating Adaptive clothing for people with disabilities to feel confident within our own skin.
Help Hope Live
Help Hope Live supports community-based fundraising for people with unmet medical expenses and related costs due to organ transplants or catastrophic injuries or illnesses. For 38 years, Help Hope Live has been showing clients and families how to bring together a network of relatives, friends, and neighbors in fundraising efforts to help cover the cost of uncovered medical expenses. These efforts play a critical role in helping our clients recover and maintain their health and independence. Since 1983, we have helped thousands of people raise millions of dollars for Help Hope Live to pay a wide range of expenses, including out-of-pocket costs for: medications, durable medical equipment, home health care, wheelchair-accessibility modifications, physical therapy, innovative treatments, medical travel and temporary relocation, even emergency living assistance. Our program has also helped thousands pay it forward and assist others with their medical expenses. Annually, we help place medical care within reach of about 5,000 families across the nation.
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.
Heart of a Giant Foundation
to eliminate preventable harm attributable to and improve the management of chronic health conditions in our communities.
My Faulty Gene
My Faulty Gene is a nonprofit organization which provides information and assistance to any individual whose family medical history suggests genetic testing might be helpful in identifying an increased risk of disease due to a genetic mutation. We believe that everyone in need of genetic testing should have access to it.
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.
Association Aux Pas du Coeur
Our organization wants to raise awareness and recognize rare diseases in Côte d'Ivoire. Our mission is to: Raising awareness and campaigning 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 so that doctors are able to make a final diagnosis and ensure the continuous follow-up of patients. Create a patient registry to establish very precise statistics of rare diseases in Côte d'Ivoire. Create a close-knit patient community. Break the isolation and despair of sick people and their families. Open up to the world and actively contribute to international research aimed at treatments.
Syndromes Without A Name (SWAN) Australia
Provide information, support and advocacy to families caring for a child with an undiagnosed or rare genetic condition.
Moonshots for Unicorns
Curing single-gene disorders
Clinical Trials
For a list of clinical trials in this disease area, please click here.