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World's priciest drug Lenmeldy aims to save rare disease lives
Lenmeldy, a one time 4.25 million infusion for metachromatic leukodystrophy, faces access hurdles as five U.S. hospitals prepare to offer the therapy. Regulators weighed price versus future gene therapies while insurers weigh coverage, access challenges, and the need for newborn screening and remote care.
MENTIONED IN THIS ARTICLE
Orchard Therapeutics
U.S
Food and Drug Administration
FDA
Meghan Halley
Stanford Center For Biomedical Ethics
Amy Gaviglio
Institute For Clinical and Economic Review
Lenmeldy
David Rind
Rind
Italy
Bobby Gaspar
James Swann
AHIP
Gaspar
Peter Marks
Center For Biologics Evaluation and Research
Celia Grace Hamlett
Minneapolis
Alabama
Celia Grace
United States
M Health Fairview Masonic Children’s Hospital
Atlanta
Philadelphia
Houston
San Francisco
Paul Orchard
Mhealth Fairview
Kassie Hamlett
Minnesota
Stephanie Keller
Children’s Healthcare of