NICE recommends life-changing gene therapy for children with ultra-rare genetic disorder in final draft guidance – Catapult’s regulatory round-up
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currently only gene therapy for children with an ultra-rare genetic disorder has been recommended by NICE in a final draft guidance published on the 23rd of March 2023.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.
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adelage (22 décembre 2023). NICE recommends life-changing gene therapy for children with ultra-rare genetic disorder in final draft guidance – Catapult’s regulatory round-up. ELSIBI. Consulté le 18 février 2026 à l’adresse https://doi.org/10.58079/vezg





