News

STAT2d
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 ...
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 ...
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
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 ...
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 ...
BioSpace1d
Sarepta Fallout Continues With EU’s Negative Opinion of Elevidys in Ambulatory Patients
The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...
BioWorld1d
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 ...