News

Sarepta fails to win EU backing for muscle disorder gene therapy

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...
Fierce Pharma2d

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 ...

EU regulator does not recommend approval of Sarepta's muscle disorder gene therapy

Europe's medicines regulator did not recommend the approval of Sarepta Therapeutics' gene therapy Elevidys for a type of rare ...
STAT3d

Why Sarepta may not survive its current gene therapy crisis

To suggest Sarepta Therapeutics may not survive its gene therapy crisis is a sobering claim to make, STAT's Adam Feuerstein ...

Sarepta Shares Fall After Gene Therapy Hits Another Regulatory Roadblock

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...

Two boys died after a gene therapy. This family won’t give up hope.

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.
STAT2d

A mother’s perspective on Sarepta’s gene therapy halt

This week on "The Readout LOUD" podcast, a mother whose son has Duchenne muscular dystrophy shares her perspective on Sarepta ...