After Sarepta reported the death of a patient who had recently taken the gene therapy Elevydis, patient advocacy group Parent ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is ...

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The initial signs of Duchenne muscular dystrophy in a 3 or 4-year-old child may be subtle, explains Dr. Alexandra ...

Duchenne muscular dystrophy (DMD) is a genetic condition that primarily affects people assigned male at birth. However, due to the condition’s X-linked inheritance pattern, only people assigned ...

Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...

If you’re living with Duchenne muscular dystrophy (DMD), planning ahead can help support the success of future education, employment, and housing efforts. Duchenne muscular dystrophy (DMD ...

Solid Biosciences will present Phase 1/2 trial data for SGT-003 gene therapy targeting Duchenne muscular dystrophy at MDA Conference. Solid Biosciences Inc. will present data from its Phase 1/2 ...

About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...

Dominic Olsen is a bright and determined 10-year-old boy living with Duchenne Muscular Dystrophy, a rare disease that has ...