The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics (NASDAQ:SRPT) gene therapy for Duchenne ...

The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics ( NASDAQ: SRPT) gene therapy for Duchenne ...

US regulators said they are investigating the death of an 8-year-old boy who received a gene therapy from Sarepta ...

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

The FDA is still on track to meet its user fee targets despite a higher-than-normal vacancy rate among scientific reviewers.

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

Elevidys, which is also facing regulatory hurdles in the US, was developed by Sarepta Therapeutics but is sold by Roche outside of the US.

Complete Response Letters recently issued by the FDA signal heightened scrutiny of trial design and reinforce the agency’s shifting regulatory expectations for sponsors and CROs.

A deep dive into the implications of the Replimune CRL that surprised insiders and investors, the role of new leadership at ...

The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...

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

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