Marina Sarzana
Milan, Italy
Last Updated: 09/26/2026
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Found 10 publications
The influence of non-surgical treatment on walking age in children with severe developmental dysplasia of the hip.
Journal: Journal of children's orthopaedics
Published: August 11, 2025
Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.
Journal: The New England journal of medicine
Published: April 23, 2025
Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndrome.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: April 02, 2025
The Italian Consensus Conference on the role of rehabilitation for children and adolescents with leukemia, central nervous system tumors, and bone cancer, part 2: general principles for the rehabilitation treatment of motor function impairments.
Journal: Pediatric hematology and oncology
Published: July 10, 2024
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Journal: Science translational medicine
Published: May 01, 2024
Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report.
Journal: Journal of personalized medicine
Published: February 22, 2023
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
Journal: The New England journal of medicine
Published: November 17, 2021
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 access.
Journal: Lancet (London, England)
Published: March 24, 2021
Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
Journal: Journal of inherited metabolic disease
Published: September 23, 2020
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
Journal: Bone marrow transplantation
Published: November 04, 2017
Showing 1-10 of 10
Last Updated: 09/26/2026