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 ...
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 ...
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 ...
Pharmaceutical Technology on MSN
Santhera grants Agamree rights to Nxera for DMD
In October 2025, Health Canada approved Agamree from Santhera for the treatment of DMD in individuals aged four years and ...
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 ...
Duchenne muscular dystrophy (DMD) is a condition that makes muscles weaker over time, specifically those you use to move, breathe, and those that help your heart function. It's caused by flaws in the ...
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