FDA approves $2.1m gene therapy for infants with rare genetic disease

The Food and Drug Administration (FDA) has issued its first approval for a gene therapy treatment targeting young children. The gene therapy is called Zolgensma, and it is designed to treat the rare genetic disease spinal muscular atrophy in children under the age of two. The FDA’s Acting Commissioner Ned Sharpless called the approval a ‘milestone’ for gene therapy. Spinal …