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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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Kaydedildi:
Asıl Yazarlar: Claire Horgan, Kelly Watts, Dipak Ram, Stewart Rust, Rebekah Hutton, Simon Jones, Rob Wynn
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Wiley 2023-09-01
Seri Bilgileri:JIMD Reports
Konular:
gene therapy
metachromatic leukodystrophy
newborn screening
transplant
Online Erişim:https://doi.org/10.1002/jmd2.12378
Etiketler: Etiketle
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