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.
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 2024
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
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.
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 is a fashion brand for the disability community. The word "chronic" in its name is a reference to people living with chronic illnesses. The fashion line is a collection of apparel items for people living with disability with clothing specially designed for men and women. The company's mission is to create, motivate, encourage, inspire, and let people know that you rock and
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.
Help Hope Live
Help Hope Live assists individuals living with catastrophic injuries and illnesses to fundraise toward their medical expenses and related costs.
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
Our mission at Jett Foundation is to extend and enrich the lives of individuals affected by Duchenne muscular dystrophy and other neuromuscular disorders.
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.
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.
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.
Clinical Trials
For a list of clinical trials in this disease area, please click here.