The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...

A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of ...

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a ...

Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an ...

Scientists at UCSF and Gladstone Institutes have identified cancer drugs that promise to reverse the changes that occur in ...

The third death occurred in an adult male with limb girdle muscular dystrophy who received a Sarepta AAVrh74 gene therapy product, SRP-9004, in a phase 4 clinical trial.

A study comparing the gene expression signature of Alzheimer's disease with those elicited by 1,300 approved drugs found a combination of two cancer medications that could treat the most common form ...

The Food and Drug Administration named a longtime pharmaceutical executive to run the agency’s drug program. Dr.

Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the ...

Sarepta Therapeutics said it will continue shipping its gene therapy Elevidys for Duchenne muscular dystrophy patients despite an FDA request to pause distribution following multiple patient deaths.

The FDA requested Sarepta voluntarily stop distributing Elevidys after the death of a third patient who received one of the firm's gene therapies.

Scientists at UC San Francisco and Gladstone Institutes have identified cancer drugs that promise to reverse the changes that ...