Posted by Michael Wonder on 23 Oct 2017
NICE approves gene therapy for rare ‘bubble baby syndrome’
23 October 2017 - Strimvelis, a treatment for an ultra-rare inherited immune deficiency condition that has been dubbed ‘bubble baby syndrome’ has been approved by NICE in draft guidance.
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 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.
