May 31, 2016

First gene therapy for children is approved in Europe: Radical treatment for rare ‘bubble boy’ disorder has a 100% survival rate

Posted by in category: biotech/medical

Great news for precision medicine.

European regulators have given the green light for a British drug firm to produce the world’s first gene therapy treatment for children.

GlaxoSmithKline was given approval by the European Commission to provide the treatment to children with a rare immune disorder — which can be fatal for those affected.

The treatment, called Strimvelis, is the first stem cell gene therapy to treat children with severe combined immunodeficiency — commonly referred to as ‘bubble boy’ syndrome.

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