New review highlights progress and cautions in AAV gene therapy for DMD
Gene therapy for Duchenne muscular dystrophy (DMD) is advancing quickly, but important safety risks still need to be carefully managed.
News
-
-
DMD worsens in young adulthood, but many report good quality of life
Arm strength and breathing get worse quickly when patients with DMD become adults, but many still report a good quality of life.
-
2026 Muscular Dystrophy Association conference highlights recent advancements
Researchers highlighted recent advances in DMD research at the Muscular Dystrophy Association’s Clinical & Scientific Conference.
-
New data on Deramiocel shows significant cardiac benefits in DMD
Dermamiocel appears to effectively prevent cardiac scarring (fibrosis) in patients with Duchenne muscular dystrophy (DMD).
-
New steroid alternative helps boys with Duchenne maintain growth
The steroid alternative vamorolone appeared safe and effective in boys with Duchenne muscular dystrophy (DMD) aged 7 to 17.
-
Gene therapy GNT0004 shows lasting benefit for boys with Duchenne
A one-time gene therapy was linked to stable or improved movement in boys with Duchenne muscular dystrophy.
-
PTC Therapeutics withdraws ataluren application after FDA review
The FDA determined that current data for ataluren in Duchenne muscular dystrophy (DMD) are unlikely to meet approval standards.
-
Genetic clues emerge for heart damage in Duchenne muscular dystrophy
Certain genes may help explain why heart disease is worse in some people with Duchenne muscular dystrophy.
-
Boys with DMD experience drop in executive function during middle years
The developmental trajectory of boys with DMD show an increase in executive function impairment with age.
-
Two-year study shows lasting gains from Elevidys gene therapy for DMD
A single gene therapy dose helped young boys with Duchenne muscular dystrophy keep their strength longer than expected.
