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

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 ...

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 ...

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

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

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

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

- ZEVASKYN is the only FDA-approved therapy to treat RDEB wounds with a single application - ...

Nanoscope Therapeutics submitted the first modules of a biologics license application for MCO-010, an investigational gene ...