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The one-time gene therapy treatment for rare, neurodegenerative disease metachromatic leukodystrophy (MLD) is the most expensive drug ever evaluated by NICE.
The 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 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 ...