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Thirty-year clinical outcomes after haematopoietic stem cell transplantation in neuronopathic Gaucher disease

BACKGROUND: Neuronopathic Gaucher Disease (nGD) describes the condition of a subgroup of patients with the Lysosomal Storage Disorder (LSD), Gaucher disease with involvement of the central nervous system (CNS) which results from inherited deficiency of β-glucosylceramidase. Although systemic manifes...

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Detalles Bibliográficos
Autores principales:	Donald, Aimee, Björkvall, Cecilia Kämpe, Vellodi, Ashok, Cox, Timothy M., Hughes, Derralyn, Jones, Simon A., Wynn, Robert, Machaczka, Maciej
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9206376/ https://www.ncbi.nlm.nih.gov/pubmed/35717194 http://dx.doi.org/10.1186/s13023-022-02378-7

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