Posted by Michael Wonder on 05 Jan 2018
NICE final draft guidance recommends gene therapy for rare ‘bubble baby syndrome’
3 January 2018 - Strimvelis, a treatment for an ultra-rare inherited immune deficiency condition that has been dubbed ‘bubble baby syndrome’ has been approved by NICE in final draft guidance.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.
