In this week’s edition of InnovationRx, we look at Moderna’s use of quantum computers, a new top drug regulator at the FDA, ...

Wall Street analysts warn that if Sarepta Therapeutics doesn't resolve the Elevidys issues, it could jeopardize its ability to repay debt in 2027.

Shares of Sarepta Therapeutics fell close to 37% on Friday after reports emerged that the US Food and Drug Administration is ...

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a muscular dystrophy patient who received a different, experimental treatment died ...

In the latest of a string of issues Sarepta has faced, the CHMP said it was recommending against approval as the pivotal ...

The FDA has come up with a series of ... some added regulatory context on troublesome issues like the added threat of adverse ... before green-lighting the drug. But with Sarepta, ...

The FDA won’t be holding an advisory panel meeting for Sarepta Therapeutics’ SRP-9001, which is now that much closer to becoming the first gene therapy for Duchenne muscular dystrophy (DMD).

Elevidys is a gene therapy approved to treat Duchenne muscular dystrophy. Last month, Sarapta halted sales of the therapy for non-ambulatory patients.

A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

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.

The FDA has finally agreed to a review a NDA for Sarepta's exon-skipping Duchenne muscular dystrophy drug eteplirsen. What's next?