A retrospective cohort study of Libmeldy (atidarsagene autotemcel) for MLD: What we have accomplished and what opportunities lie ahead

A retrospective cohort study of Libmeldy (atidarsagene autotemcel) for MLD: What we have accomplished and what opportunities lie ahead

Abstract Metachromatic leukodystrophy (MLD) results from ARSA gene mutations. Affected individuals meet early milestones before neurological deterioration and early death. Atidarsagene autotemcel (arsa‐cel), an autologous haematopoietic stem cell gene therapy (HSC‐GT) product, has demonstrated susta...

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Bibliographic Details
Main Authors: Claire Horgan, Kelly Watts, Dipak Ram, Stewart Rust, Rebekah Hutton, Simon Jones, Rob Wynn
Format: Article
Language:English
Published: Wiley 2023-09-01
Series:JIMD Reports
Subjects:
gene therapy
metachromatic leukodystrophy
newborn screening
transplant
Online Access:https://doi.org/10.1002/jmd2.12378

Internet

https://doi.org/10.1002/jmd2.12378

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