FDA Grants Breakthrough Therapy Designation to Gene Therapy for Treatment of CALD – Rare Disease Report
Rare Disease Report |
FDA Grants Breakthrough Therapy Designation to Gene Therapy for Treatment of CALD
Rare Disease Report The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to bluebird bio, Inc.'s Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy (CALD), a serious X-linked genetic disease that is rare and … |
