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 ...

MPS IIIA is caused by mutations in the SGSH gene, leading to a deficiency of the enzyme heparan N-sulfatase, causing toxic accumulation of heparan sulfate in the brain and body. It causes rapid ...

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 ...

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

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