Duchenne muscular dystrophy

News

Despite trial miss, Sarepta plans new filing for DMD therapy

Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy has failed to meet its primary objective in a phase 3 trial, but the company says it will file to extend

News

Santhera closes on first approval for DMD drug vamorolone

Swiss biotech Santhera is on the brink of its first regulatory approval for Duchenne muscular dystrophy treatment vamorolone, after getting a positive opinion on the drug

News

EMA starts review of Italfarmaco's Duchenne drug

Italfarmaco's marketing application for HDAC inhibitor givinostat as a treatment for Duchenne muscular dystrophy (DMD) has been accepted by the EMA, setting up an approval

News

FibroGen slumps on second failed DMD study

FibroGen has revealed that a second late-stage clinical trial of Duchenne muscular dystrophy (DMD) therapy pamrevlumab has failed to show efficacy, extending a run of bad

News

EMA backs SV95C as digital primary endpoint for DMD trials

The EMA has endorsed a digital measurement using wearables developed by Sysnav Healthcare as a primary endpoint for use in trials involving patients with Duchenne muscular

News

Big day for Duchenne as Sarepta gets first gene therapy nod

Almost against the odds, Sarepta’s gene therapy for muscle-wasting disease Duchenne muscular dystrophy (DMD) has been given accelerated approval by the FDA, albeit in a fa

Despite trial miss, Sarepta plans new filing for DMD therapy

Santhera closes on first approval for DMD drug vamorolone

EMA starts review of Italfarmaco's Duchenne drug

FibroGen slumps on second failed DMD study

EMA backs SV95C as digital primary endpoint for DMD trials

Big day for Duchenne as Sarepta gets first gene therapy nod

GSK makes fresh neuroscience foray with £2bn+ ABL deal

Health Trends 2025: Reimagining What's Possible

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Duchenne muscular dystrophy

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