health-gene-therapy2.02 min read "Record-breaking gene therapy priced at $4.3 million approved for rare genetic disease in the US"
A new gene therapy treatment, priced at $4.25 million, has already transformed the lives of children with a fatal neurodegenerative disease called metachromatic leukodystrophy (MLD). Addi and Oliver Rasberry, both born with the same genetic mutation causing MLD, have had vastly different experiences due to the availability of the gene therapy. While Addi struggles with daily medical interventions, Oliver, who received the gene therapy in Milan, is thriving. The therapy, manufactured by Orchard Therapeutics and approved in Europe in 2021, will soon be available in the U.S., offering hope to families affected by MLD.
1 year ago•Source: STAT
