Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-shot treatment.
Orchard Therapeutics' gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The agency said that while there ...
It appears that Libmeldy has provided a permanent fix for his MLD. "It's given him his life back. He makes the most of every moment," says his mum, Nicola. Joe Elson had gene therapy for MLD in ...