Duchenne muscular dystrophy (DMD) causes muscle weakness that becomes more severe over time. While there is no cure, treatments can help slow the progression and manage symptoms and complications.

Capricor Therapeutics is advancing deramiocel (CAP-1002) for DMD cardiomyopathy, with a BLA resubmission and HOPE-3 topline data expected by 2026. CAPR's therapy addresses a critical unmet need in non ...

Duchenne muscular dystrophy (DMD) occurs as a result of genetic changes on the X chromosome. If someone has a gene change that can cause DMD, their children may inherit that change. DMD is a ...

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...

The FDA has rejected Capricor Therapeutics’ filing for approval of a Duchenne muscular dystrophy (DMD) cell therapy, raising questions about whether the agency's new leadership may be rowing back from ...

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing ...

A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. Sarepta and its ex-U.S ...

Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys ® (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients following the second death in three months of ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Edgewise Therapeutics' valuation is heavily reliant on DMD/BMD programs, with upcoming phase-2 readouts likely to disappoint and trigger a sharp stock decline. We believe that sevasemten's mechanism ...