After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
Keros Therapeutics KROS is a clinical-stage biopharmaceutical company developing innovative therapies for patients with ...
CDMO giant Catalent inked a deal with Sarepta Therapeutics to manufacture what looks to become the first gene therapy to treat Duchenne muscular dystrophy (DMD). In November, the FDA gave its ...
Sarepta Therapeutics SRPT announced positive three-year top-line data from Part 1 of the phase III EMBARK study, evaluating ...
After escaping a clinical hold several years back, Dyne Therapeutics has revealed new phase 1/2 data for its Duchenne muscular dystrophy (DMD) therapy DYNE-251. The readout notes several serious ...
KROS is advancing KER-065 for Duchenne muscular dystrophy, with orphan drug status and a phase II trial planned for early 2026.
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered acute ...
Taiho Pharmaceutical’s investigational treatment for Duchenne muscular dystrophy (DMD) has failed to impact the time it takes patients to rise from the floor over 52 weeks, missing the phase 3 study’s ...
Sarepta Therapeutics said it will update its prescribing information for Elevidys ® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...
Dyne Therapeutics leverages its FORCE platform to target genetically driven muscle diseases. Read why I am bullish about DYN ...