NICE approves gene therapy for rare Bubble Baby Syndrome

Children with ADA-SCID, severe combined immunodeficiency due to adenosine deaminase deficiency, are extremely vulnerable to infection and usually live in isolation to minimise the risk, hence the nickname ‘baby in a bubble’. Up until now the only treatment has been a stem cell transplant but these are risky and it is not always possible to find a good match.

Now NICE (National Institute for Health and Care Excellence) has approved a new treatment for those children who cannot find a good match. It could mean children with ADA-SCID have the chance of going to school and socialising without the constant fear of catching a simple infection that could prove life threatening.

The full article is available at the NICE site:

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