Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing them to weaken and deteriorate over time. While muscular dystrophies are often diagnosed in childhood, some types appear in adulthood.

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North Star Ambulatory Assessment,

Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory patients with Duchenne muscular dystrophy following two deaths caused by acute liver failure.

The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, has so far declined to begin the treatment.

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer technologies,

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy,

What Happened? Shares of biotech company Sarepta Therapeutics (NASDAQ:SRPT) fell 44.4% in the afternoon session after the company reported a second death linked to its Duchenne muscular dystrophy gene therapy,

A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys.