Posted by Michael Wonder on 04 Feb 2022
First gene therapy for rare genetic neurodegenerative disorder in children, recommended in NICE draft guidance
4 February 2022 - Children with a rare, fatal, genetic disorder will be able to benefit from a new one-off treatment under new NICE guidance.
Gene therapy atidarsagene autotemcel (Libmeldy, Orchard Therapeutics) will be available for some children with the rare, life-limiting inherited neurodegenerative condition metachromatic leukodystrophy.
