Evaluating meaningful changes in physical functioning and cognitive declines in metachromatic leukodystrophy: a caregiver interview study

BACKGROUND: Metachromatic leukodystrophy (MLD) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase A (ASA). Treatment options for patients are limited; gene therapy based on haematopoietic stem cell transplantation is the only approved treatment for some subtypes of MLD...

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Detalles Bibliográficos
Autores principales: Martin, Susan, Harris, Nimanee, Romanus, Dorothy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10352216/
https://www.ncbi.nlm.nih.gov/pubmed/37458805
http://dx.doi.org/10.1186/s41687-023-00595-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10352216/
https://www.ncbi.nlm.nih.gov/pubmed/37458805
http://dx.doi.org/10.1186/s41687-023-00595-7

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