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Gene therapy for 'bubble baby' syndrome approved on NHS

GlaxoSmithKline’s Strimvelis is the first such treatment to be funded in the UK

The NHS will fund gene therapy for the first time after the UK’s healthcare cost watchdog approved treatment for the so-called “bubble baby” syndrome, despite a price tag of more than £500,000.

The treatment is used against adenosine deaminase deficiency, or ADA-SCID, which disables the immune system and means that children with the illness have to be kept in isolation to avoid infection – hence the “bubble baby” name.

Continue reading... October 23, 2017 at 07:21PM

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