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October 24, 2017

Britain backs GSK's gene therapy for 'bubble boy' syndrome

  • Government
  • Agency
  • Industry
  • Genetics/Genomics

Reuters – GlaxoSmithKline’s gene therapy for the so-called “bubble boy” disease was approved by Britain’s healthcare cost watchdog NICE, despite a price tag of almost 600,000 euros ($700,000). […]

Britain’s National Institute for Health and Care Excellence (NICE) said in draft guidance published on Friday that Strimvelis gene therapy used against adenosine deaminase deficiency, or ADA-SCID, improves overall survival compared with standard stem cell transplant therapy.

The inherited condition disables the immune system and without treatment, children with ADA-SCID need to be kept in isolation to avoid infections – hence it has become known as the “bubble baby” or “bubble boy” syndrome.

Read more at Reuters.