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 ...

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

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 ...

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 ...

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

REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...

The two brothers have Duchenne muscular dystrophy — a rare, inherited muscle-wasting disorder that has no cure. "Caleb stopped walking in December of '23, so a little over a year ago.

Newborns in Minnesota can now be screened for Duchenne muscular dystrophy (DMD) and guanidinoacetate methyltransferase (GAMT) ...

Solid Biosciences said Tuesday that the first three patients to receive its experimental gene therapy for Duchenne muscular dystrophy all produced high levels of microdystrophin, a miniature ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...

Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...