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Murky road ahead for Duchenne gene therapy as confirmatory trial misses main goal

When the first gene therapy for the rare, debilitating disease Duchenne muscular dystrophy was conditionally approved earlier this year, it was hailed as a seminal moment for patients and their families an opportunity to try to slow the progression of a disease that robs kids of their ability to walk by their teen years, and can be fatal by age 30.

Doug ingramDrug administrationSarepta therapeuticsJohns hopkinsNorth star ambulatory assessment

Sarepta s gene therapy Elevidys misses primary goal

Sarepta Therapeutics has hit a setback as its approved gene therapy Elevidys missed the primary goal of a phase 3 trial in children aged 4 through 7 years with the inherited disease Duchenne muscular dystrophy (DMD).

Doug ingramDrug administrationNorth star ambulatory assessmentCell amp gene therapiesBio developmentsEmerging marketsDuchenne muscular dystrophyGene therapySarepta therapeutics

Why Sarepta Therapeutics Stock Is Crashing Today

There's a plot twist with Sarepta's disappointing clinical results.

Doug ingramDrug administrationSarepta therapeuticsNorth star ambulatory assessmentAlthough elevidys

Sarepta s Duchenne gene therapy fails primary endpoint, but company presses on

Sarepta Therapeutics’ Elevidys failed to meet its primary endpoint in a Phase 3 trial, shortly after the FDA granted it accelerated approval for Duchenne muscular dystrophy.

Sarepta elevidysDoug ingramDrug administrationSarepta therapeuticNorth star ambulatory assessment

Murky road ahead for Duchenne gene therapy as confirmatory trial misses main goal

Murky road ahead for Duchenne gene therapy as confirmatory trial misses main goal
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Doug ingramDrug administrationSarepta therapeuticsJohns hopkinsNorth star ambulatory assessment

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