News

Sarepta Therapeutics Provides Clarifying Statement on ELEVIDYS

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the ...
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 ...

FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...

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 ...

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.
STAT1d

For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
BioPharma Dive22h

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 ...

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 ...