News

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 opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
GEN - Genetic Engineering and Biotechnology News9h
FDA Probes Death of Brazilian Boy Linked to Sarepta’s Elevidys
The FDA is investigating the death last month of an eight-year-old boy in Brazil who had received Sarepta Therapeutics’ Elevidys® (delandistrogene moxeparvovec-rokl)—though the company and its ...
Fierce Biotech2d
'We will crack gene therapy': Elevidys fallout doesn't dent Roche's hopes for modality
Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...
Fierce Pharma3d
FDA weighs new study requirements for Sarepta to confirm safety of gene therapy Elevidys: report
Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...
STAT2d
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 Dive2d
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 ...
Fierce Pharma2d
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 ...
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 ...