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SCID Treatment Funded in UK

The UK's National Institute for Health and Care Excellence (NICE) has approved GlaxoSmithKline's gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), Reuters reports.

For GSK's Strimvelis, patients' CD34+ cells are reprogrammed to express ADA to treat the condition. Patients with ADA-SCID are kept in isolation to protect them from infections. The European Commission approved the therapy, which is to be a one-time treatment, in 2016.

Reuters notes that the UK's NICE approved the therapy despite its cost of nearly €600,000 (US$700,000). As the Times reports, NICE has placed a cap on what it would pay for treatments for rare diseases, saying that it wouldn't pay for therapies that topped £100,000 for a year of good-quality life. But the agency estimates that the treatment gives 14 years of good-quality life, bringing its cost under that limit.

 "It's an extremely exciting time for gene therapy," Bobby Gaspar, a researcher at Great Ormond Street, tells the Times

"We are getting to the point where we have a number of standard genetic medicines and that will only encourage the development of others," he adds.

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