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A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel

OBJECTIVE: To understand the benefit-risk profile for historical and current treatments for MLD. METHODS: A systematic review was conducted on the effectiveness, safety, and costs of MLD treatments: allogeneic haematopoietic stem cell transplantation (HSCT) and atidarsagene autotemcel (arsa-cel) acc...

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Detalles Bibliográficos
Autores principales:	Armstrong, Nigel, Olaye, Andrew, Noake, Caro, Pang, Francis
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10466877/ https://www.ncbi.nlm.nih.gov/pubmed/37644601 http://dx.doi.org/10.1186/s13023-023-02814-2

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