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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Hlavní autoři:	Claire Horgan, Kelly Watts, Dipak Ram, Stewart Rust, Rebekah Hutton, Simon Jones, Rob Wynn
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Wiley 2023-09-01
Edice:	JIMD Reports
Témata:	gene therapy metachromatic leukodystrophy newborn screening transplant
On-line přístup:	https://doi.org/10.1002/jmd2.12378
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