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The New England Journal of Medicine|November 17, 2021
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler SyndromeBernhard Gentner, Francesca Tucci, Stefania Galimberti, et al.
Molecular Genetics and Metabolism|July 4, 2024
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophyLaura Ann Adang, Samuel Groeschel, Chloe Grzyb, et al.
Lancet (London, England)|January 23, 2022
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded accessFrancesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, et al.
The New England Journal of Medicine|April 23, 2025
Long-Term Effects of Atidarsagene Autotemcel for Metachromatic LeukodystrophyFrancesca Fumagalli, Valeria Calbi, Vera Gallo, et al.
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