The FDA approved labeling changes for elivaldogene autotemcel (eli-cel; Skysona), a one-time gene therapy for patients with early, active cerebral adrenoleukodystrophy (CALD), to reflect new safety ...

Neuroblastoma can be a particularly insidious cancer. In about half of all cases, tumors regress, even without therapy. In ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona ...

Neuroblastoma, a cancer mainly affecting children, is often difficult to treat. A team led by Jan Dörr and Anton Henssen at ...

Net loss per share (GAAP) was $0.62, but remained negative as the company continues to invest heavily in research and development. The pivotal Phase 2 trial for Danon disease was placed on clinical ...

Sarepta Therapeutics Inc.’s shares soared after the US biotech reported revenue that beat estimates, which analysts ...

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

The FDA's approval of the first CRISPR-Cas9–based gene therapy marked a major milestone in biomedicine, validating genome ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with ...