Posted by Michael Wonder on 25 Mar 2023
NICE recommends life-changing gene therapy for children with ultra rare genetic disorder in final draft guidance
23 March 2023 - The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended by NICE in final draft guidance published today.
It is estimated there are around 10 children with the condition, called aromatic L-amino acid decarboxylase (AADC) deficiency, in the UK, of who a small number may be eligible for treatment with eladocagene exuparvovec.
