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European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|March 30, 2024
Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical managementLucia Laugwitz, Daphne H Schoenmakers, Laura A Adang, et al.
Lancet (London, England)|June 13, 2016
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trialMaria Sessa, Laura Lorioli, Francesca Fumagalli, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 29, 2025
Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndromeFrancesca Tucci, M Luz Uria Oficialdegui, Giulia Consiglieri, et al.
Bone Marrow Transplantation|June 1, 2019
Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapyFrancesca Tucci, Marta Frittoli, Federica Barzaghi, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|July 10, 2026
European expert recommendations for comprehensive pre-treatment, treatment-phase and post-treatment care of patients with metachromatic leukodystrophy treated with autologous haematopoietic stem and progenitor cell gene therapyLucia Laugwitz, Francesca Fumagalli, Katharina Wehner, et al.
Cytotherapy|April 13, 2024
Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United StatesLaura A Adang, Joshua L Bonkowsky, Jaap Jan Boelens, et al.
Neurology|June 27, 2025
Metachromatic Leukodystrophy: New Therapy Advancements and Emerging Research DirectionsMarije A B C Asbreuk, Daphne H Schoenmakers, Laura Ann Adang, et al.
Science (New York, N.Y.)|July 13, 2013
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophyAlessandra Biffi, Eugenio Montini, Laura Lorioli, et al.
Science Translational Medicine|May 1, 2024
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndromeGiulia Consiglieri, Francesca Tucci, Maurizio De Pellegrin, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|June 7, 2025
Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophyDaphne H Schoenmakers, Marije A B C Asbreuk, Tamara Martin, et al.
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