Dyne Therapeutics wins FDA Priority Review for DMD drug z-rostudirsen, with key catalysts and pipeline upside. Click for more ...

Priority Review granted; PDUFA target action date set for January 21, 2027 -- Submission for Accelerated Approval based on dystrophin as a ...

In the open-label study, DMD patients received Satellos’ oral drug for an additional 11 months, following an earlier 28-day trial.

Precision BioSciences has activated Arkansas Children’s Hospital as the first site and initiated patient enrolment for its Phase I/II FUNCTION-DMD clinical trial of PBGENE-DMD for Duchenne muscular ...

Researchers at the USF Health Morsani College of Medicine have identified a potential pathway that could protect cardiac ...

Phase 3 EPIDYS MRI data quantify givinostat’s potential to preserve functional muscle tissue and reduce fat infiltration in ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

In yet another fail for the Duchenne muscular dystrophy (DMD) field, Taiho Pharmaceutical Co. Ltd.’s pizuglanstat (TAS-205) did not meet the primary endpoint in a phase III trial. The phase III ...

-Arkansas Children’s Hospital activated as the first clinical trial site and now enrolling patients in the FUNCTION-DMD study of PBGENE-DMD- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical ...