NICE recommends life-changing gene therapy for children with ultra-rare genetic disorder in final draft guidance - Catapult's regulatory round-up
Fiche du document
22 décembre 2023
- doi: 10.58079/vezg
info:eu-repo/semantics/openAccess
Sujets proches
Childhood Youngsters Pedology (Child study) Kids (Children)Citer ce document
adelage, « NICE recommends life-changing gene therapy for children with ultra-rare genetic disorder in final draft guidance - Catapult's regulatory round-up », ELSIBI, ID : 10.58079/vezg
Métriques
Partage / Export
Résumé
The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended by NICE in a final draft guidance published on the 23rd of March 2023. Children with the condition, called aromatic L-amino acid decarboxylase (AADC) deficiency, in the UK, may be now eligible for treatment with eladocagene exuparvovec. Published on the 3rd of April 2023 in Catapult's regulatory round-up for March 2023.