Antisense therapy with both chemistries of phosphorodiamidate morpholino oligomers (PMOs) and 2′-O-methyl phosphorothioate has demonstrated the capability to induce dystrophin expression in Duchenne ...

Capricor Therapeutics has presented positive long-term data from its HOPE-2 clinical trial at the 2025 Muscular Dystrophy Association Conference, indicating that its leading treatment, deramiocel, can ...

Treatment with deramiocel led to a 54% slowing of upper limb function deterioration and a 91% slowing of cardiac function deterioration compared with placebo. Topline data were announced from a phase ...

(RTTNews) - Capricor Therapeutics, Inc. (CAPR), on Monday, announced positive data from its ongoing HOPE-2 open-label extension trial, demonstrating the long-term efficacy of Deramiocel for the ...

A new surprise discovery could hold big implications for muscular dystrophy patients, with researchers finding that an existing cancer drug may delay progression of the hard-to-treat disease.

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new positive efficacy and safety results showing ...

Edasalonexent has been shown to preserve muscle function and substantially slow DMD disease progression across all four assessments of muscle function (the North Star Ambulatory Assessment, time to ...

Capricor Therapeutics’ Duchenne muscular dystrophy therapy (DMD) appears to have slowed disease progression in older boys and young men who are in the later stages of their disease and unable to walk, ...

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. If efficiently and safely scaled up ...

Using a new gene-editing technique, a team of scientists has stopped progression of Duchenne muscular dystrophy in young mice. DMD, the most common and severe form of muscular dystrophy among boys, is ...

-- Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and ...