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Following US FDA approval of Lenmeldy™ (atidarsagene autotemcel) for early-onset metachromatic leukodystrophy (MLD), the US wholesale acquisition cost of the gene therapy has been set to $4.25 million.
The one-time gene therapy treatment for rare, neurodegenerative disease metachromatic leukodystrophy (MLD) is the most expensive drug ever evaluated by NICE.
11 May 2016 | By Victoria White, Digital Content Producer
University of Manchester and CMFT researchers are to work with Orchard Therapeutics to test a stem cell gene therapy for Sanfilippo disease in a human trial...
