A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

Sarepta and Roche announced a 16-year-old US patient dosed with the gene therapy died of acute liver failure in March 2025.

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

New research identifies the body's own immune response as a critical and previously underappreciated barrier to successEarly intervention at birth ...

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

The Swiss drugmaker, which holds European rights to Elevidys, suspended three trials while researchers investigate the death ...

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

Roche Holdings AG (OTC:RHHBY) on Thursday presented new data at the AD/PD 2025 International Conference on Alzheimer’s and ...

Oppenheimer analyst Andreas Argyrides lowered the firm’s price target on Sarepta (SRPT) to $184 from $215 and keeps an Outperform rating on the ...

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

In March, Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.