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NICE recommends first treatment for children with metachromatic leukodystrophy
February 09, 2022The one-time gene therapy treatment for rare, neurodegenerative disease metachromatic leukodystrophy (MLD) is the most expensive drug ever evaluated by NICE. -
NICE ‘no’ for Orchard Therapeutics’ gene therapy Libmeldy
July 13, 2021The National Institute for Health and Care Excellence (NICE) has announced that it does not recommend Orchard Therapeutics’ gene therapy Libmeldy for the treatment of metachromatic leukodystrophy (MLD) in children. -
Orchard Therapeutics Announces OTL-200 Granted RMAT Designation
January 21, 2021Orchard Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the ...
Pharma Sources Insight January 2025
