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

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...

ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...

Ifetroban demonstrated significant 5.4% improvement in cardiac functionNASHVILLE, Tenn., March 19, 2025 /PRNewswire/ -- Cumberland Pharma ...

Announced the U.S. FDA has accepted our Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne ...

Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular ...

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...