Gene Therapy for Degenerative Brain Disorder Wins FDA Nod

(MedPage Today) -- 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...
Source: MedPage Today Pediatrics - Category: Pediatrics Source Type: news