Gene Therapy for Degenerative Brain Disorder Wins FDA Nod
The FDA granted accelerated approval to elivaldogene autotemcel (eli-cel; Skysona), a gene therapy to treat early, active cerebral adrenoleukodystrophy (CALD) in boys ages 4 to 17 years, drugmaker bluebird bio announced Friday. Childhood CALD is a rare, neurologically debilitating form of adrenoleukodystrophy that generally occurs in young boys. It is caused by a mutation in […]