Shortcast: A retrospective cohort study of Libmeldy (atidarsagene autotemcel) for MLD
Oct 19, 2023•5 min
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Episode description
Dr Claire Horgan discusses the first year of offering gene therapy to patients with metachromatic leukodystrophy (MLD) in the UK. When given in a timely fashion the impact is incredible but large numbers of children remain ineligible for treatment.
A retrospective cohort study of Libmeldy (atidarsagene autotemcel) for MLD: What we have accomplished and what opportunities lie ahead
Claire Horgan, et al
https://doi.org/10.1002/jmd2.12378
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