Delandistrogene moxeparvovec-rokl shows significant motor function improvements in 8- and 9-year-olds with DMD, countering expected motor decline. Statistically significant differences were observed ...

Biogen’s salanersen is now heading into three Phase III trials in SMA.

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Four patients with LGMD-R5 (gamma-sarcoglycanopathy, formerly LGMD-2C) have received the ATA-200 gene therapy as part of the ongoing Phase 1b/2 trial evaluating the safety, pharmacodynamics, and ...

ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the ...

Researchers developed a gene therapy for LAMA2-related muscular dystrophy that restored muscle and nerve function in mice.

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...

When Harith Rajagopalan considers the millions of patients who have taken a GLP-1 drug to treat diabetes or obesity, he sees a revolution that is failing to realize its promise. “We are literally ...

When Domenic Catrine was born, his heel swelled up from his vitamin K shot. His circumcision site wouldn't stop bleeding. At the Children's Hospital of Philadelphia, Catrine was diagnosed as having ...