Beur schreef op 30 november 2023 14:30:
Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH
Previously reported results from ongoing proof-of-concept study showed extensive metabolic correction, continued cognitive, motor, and physical development, as well as early improvements in skeletal health
Additionally, study investigators presented favorable outcomes for other disease manifestations not effectively addressed by the current standard of care at ESGCT 2023
Global registrational trial expected to commence by year-end
ir.orchard-tx.com/news-releases/news-...=================================
Orchard Theraputics ontwikkelt voor Pharming OTL-105