"Record-breaking gene therapy priced at $4.3 million approved for rare genetic disease in the US"
TL;DR Summary
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.
Topics:business#childrens-health#gene-therapy#genetic-mutation#health-gene-therapy#medical-breakthrough#neurodegenerative-disease
- New gene therapy, to be priced at $4.25 million, has already transformed children's lives STAT
- A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access CNN
- Kyowa Kirin's gene therapy most expensive US drug with $4.3 million price tag Yahoo Finance
- After Kyowa Kirin buyout, Orchard scores FDA approval for first MLD gene therapy in the US FiercePharma
- US approves first gene therapy for children with rare genetic disease Reuters
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