News

FDA Probes Death of Boy in Brazil on Sarepta’s Elevidys

US regulators are investigating the death of an 8-year-old boy in Brazil who received Sarepta Therapeutics Inc.’s Elevidys.

Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...
STAT1d

Europe moves to reject embattled Duchenne muscular dystrophy gene therapy

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
BioPharma Dive1d

Sarepta woes mount as Duchenne gene therapy knocked back in Europe

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
Fierce Pharma1d

Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe

Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...

Health Beat: Duchenne muscular dystrophy

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...