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

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 sustained clin...

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Detalles Bibliográficos
Autores principales:	Horgan, Claire, Watts, Kelly, Ram, Dipak, Rust, Stewart, Hutton, Rebekah, Jones, Simon, Wynn, Rob
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2023
Materias:	Research Reports
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10494509/ https://www.ncbi.nlm.nih.gov/pubmed/37701322 http://dx.doi.org/10.1002/jmd2.12378

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