Patients in Scotland can't get the treatment - which is available in England - despite manufacturers giving it away for free ...

The firm said the clinical trial pause is temporary as an independent data monitoring committee finalizes its analysis for submission to regulators.

Some studies of Sarepta Therapeutics' muscular dystrophy drug, Elevidys, have been halted by European regulators after a ...

Therapeutics shared the following update related to ELEVIDYS, the only approved gene therapy in patients with Duchenne muscular ...

Sarepta Therapeutics said on Friday it had temporarily halted three trials testing its gene therapy Elevidys, following the death of a patient last month upon receiving the treatment.

Shares of Sarepta Therapeutics SRPT dropped more than 6% yesterday after the EMA placed a clinical hold on all studies ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

BioMarin Pharmaceutical Inc. has advancements in phenylketonuria treatment. Click to learn how Palynziq's success could ...

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...

The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US ...