FDA Grants Breakthrough Therapy Designation to Gene Therapy for Cerebral Adrenoleukodystrophy – AJMC.com Managed Markets Network
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FDA Grants Breakthrough Therapy Designation to Gene Therapy for Cerebral Adrenoleukodystrophy
AJMC.com Managed Markets Network The FDA has granted a Breakthrough Therapy designation to bluebird bio, Inc's Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy (CALD), an X-linked genetic disorder caused by a defect in the gene ABCD1. This mutation results in … |
