“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). SCID is sometimes known as “bubble baby” disease, since those born with it have immune systems so weak they must live in germ-free environments.
Strimvelis is the second gene therapy to be approved in Europe, after UniQure’s Glybera, which treats a rare adult blood disorder.”
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