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Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease
Krabbe disease is a rare neurodegenerative disorder caused by a deficiency in galactocerebrosidase. The only effective treatment is hematopoietic stem cell transplantation (HSCT). Approximately 85% of Krabbe disease cases are the infantile subtypes, among which ∼20% are late infantile. Prior studies...
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| Publicado no: | Blood |
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| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8020262/ https://ncbi.nlm.nih.gov/pubmed/33150395 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood.2020005477 |
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