Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report

(1) Background: Atidarsagene autotemcel is a hematopoietic stem and progenitor cell gene therapy (HSPC-GT) approved to treat early-onset metachromatic leukodystrophy (MLD). The purpose of this case report is to describe the long-term management of residual gait impairment of a child with late infant...

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Detalles Bibliográficos
Autores principales: Faccioli, Silvia, Sassi, Silvia, Pandarese, Daniela, Borghi, Corrado, Montemaggiori, Valentina, Sarzana, Marina, Scarparo, Stefano, Butera, Carla, Calbi, Valeria, Aiuti, Alessandro, Fumagalli, Francesca
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10144348/
https://www.ncbi.nlm.nih.gov/pubmed/37109023
http://dx.doi.org/10.3390/jpm13040637

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10144348/
https://www.ncbi.nlm.nih.gov/pubmed/37109023
http://dx.doi.org/10.3390/jpm13040637

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