GSK gets EU approval for first gene therapy for children
The world's first life-saving gene therapy for children, developed by GlaxoSmithKline and Italian scientists, was approved for sale in Europe on Friday, in a step forward for the pioneering technology to fix faulty genes. Called Strimvelis, it is designed for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID). Strimvelis is the second gene therapy to be approved in Europe, after UniQure&039;s Glybera, which treats a rare adult blood disorder.
