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Genetic and Metabolic :
Muscular Dystrophy / Duchenne Muscular Dystrophy

Muscular dystrophy (MD) refers to a group of hereditary disorders which causes muscles to weaken and waste away. There is no cure for MC, but drug treatment for symptoms, therapy, and assistive technology can make a huge difference for a person with the condition.

Learn more about muscular dystrophy from doctors and other people and families dealing with the condition in this online support group for MD.

 

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Gene Therapy Offers New Hope Duchenne Muscular Dystrophy Patients
Researchers have achieved a significant breakthrough in developing a new gene therapy approach that restores the full-length dystrophin prote.....
ELEVIDYS Gene Therapy Shows Promise Duchenne Muscular Dystrophy
Ann & Robert H.Lurie Children’s Hospital of Chicago treated its first Duchenne muscular dystrophy patient on March 27, 2024with ELEVIDYS (received U.S.....
New Chapter for Duchenne Muscular Dystrophy Treatment
FDA's endorsement of vamorolone, an innovative anti-inflammatory medication created in collaboration with Children's National, offers a promising, low.....
Mitochondrial Pore: A Potential Game-Changer for Muscular Dystrophy Treatment
Mitochondria, the powerhouse of the cell helps prevent the muscle-wasting symptoms of muscular dystrophy (MD). T.....
FDA Approves Gene Therapy for Deadly Form of Muscular Dystrophy
Elevidys, the first gene therapy for treating the rare and deadly muscle disorder Duchenne muscular dystrophy (DMD) has been approv.....


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