Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other parents still push for the therapy.

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s focus in neuromuscular diseasesCapricor remains on track for the August 31,

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug Administration granted the special status to Deramiocel, the company's lead cell therapy candidate.

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.

The boys live in Reed City with their mother Tabatha; father Michael, who is the Osceola County Road Commission manager and sister Elizabeth, 11. They were diagnosed with Duchenne muscular dystrophy in 2015. Their mother, Tabatha Mattzela recounted finding out.

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.

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,