Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy
Article 2025 en
Authors
AC
Alessandra Clerici
AC
Ambra Corti
MF
Marcella Facchini
Abstract
1 min read
Among patients with presymptomatic late-infantile or early-juvenile MLD and those with early-symptomatic early-juvenile MLD, the risk of severe motor impairment or death was significantly lower among those who received treatment with arsa-cel than in a natural history cohort that did not receive treatment. (Funded by Orchard Therapeutics and others; ClinicalTrials.gov numbers, NCT01560182 and NCT03392987.).
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Meletios A Dimopoulos, K. Weisel, Kevin Song, Michel Delforge, Lionel Karlin, Hartmut Goldschmidt, Philippe Moreau, A. Baños, Albert Oriol, L. Garderet, Michèle Cavo, V. L. Ivanova, Adrían Alegre, Joaquín Martínez‐López, Christopher Chen, Andrew Spencer, Stefan Knop, Nizar J. Bahlis, Christoph Renner, Xin Yu, Kevin Hong, Lars Sternås,
Raymond A. van Adrichem, Banne Nemeth, Ale Algra, Saskia le Cessie, Frits R. Rosendaal, Inger B. Schipper, Rob G. H. H. Nelissen, Suzanne C. Cannegieter
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