This study marks the first time gene therapy has shown consistent success in both children and adults with OTOF-related ...

Sarepta Therapeutics faces a challenging period as it navigates recent setbacks with its groundbreaking gene therapy, leading ...

An expert discusses how recent updates to AUA, EAU and NCCN guidelines have cautiously shifted toward more bladder-sparing approaches for BCG-unresponsive patients, incorporating new FDA-approved ...

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

An expert discusses how high-cost gene therapies require evidence standards including Phase 3 trial data showing complete response rates and durability, real-world evidence and health economic ...

Sarepta Therapeutics Inc. is cutting more than one-third of its workforce and will add a black box warning label to its gene therapy for a fatal muscle disorder after two patient deaths raised doubts ...

Sarepta Therapeutics said on Wednesday it would lay off 36% of its workforce, or about 500 employees, after the recent deaths of two patients who had received its gene therapy Elevidys.

The company is also pausing research for several treatments it has been developing for another form of muscular dystrophy.

Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

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

The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...