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Gene Therapy For Children Approved In Europe

In a global first, gene therapy that could prove life-saving for children has been recommended for approval in Europe. The pioneering technology to fix faulty genes has been developed by Italian scientists and GlaxoSmithKline FOX News reports: The European Medicines Agency (EMA) said on Friday it had endorsed the therapy, called Strimvelis, for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no matching bone marrow donor is available. Around 15 children a year are born in Europe with the ultra-rare genetic disorder, which leaves them unable to make a type of white blood cell. They rarely survive beyond two years unless their immune function is restored with a suitable bone marrow transplant. SCID is sometimes known as “bubble baby” disease, since children born with it have immune systems so weak they must live in germ-free environments. Strimvelis is expected to secure formal marketing authorization from the European Commission in a couple of months, making it the second gene therapy to be approved in Europe, after UniQure’s Glybera, which treats a rare adult blood disorder. The U.S. Food and Drug Administration has yet to approve any gene therapies but a growing number of U.S. biotech [...]