News

Sarepta denies Elevidys is linked to latest patient death in Brazil
Shortly after the U.S. FDA announced an investigation into the death of a patient who had received Elevidys, a gene therapy ...
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 Dive23h
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 ...
BioWorld17h
CHMP rejects Sarepta’s Elevidys, changes mind on Lilly’s Kisunla
After a tough few weeks for Sarepta Therapeutics Inc., the EMA dealt another blow on July 25, announcing it will not be ...
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 ...