Improved Brain Pathology and Progressive Peripheral Neuropathy in a 15 Year Old Survivor of Infantile Krabbe Disease Treated With Umbilical Cord Transplantation

ObjectiveKrabbe disease is a fatal leukodystrophy caused by deficiency in galactocerebrosidase enzyme activity. The only currently available therapy is hematopoietic stem cell transplantation with bone marrow or umbilical cord blood (UCBT), which leads to increased lifespan and functional abilities...

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Principais autores:	Julia Kofler, Maria L. Beltran-Quintero, Anne Rugari, Giulio Zuccoli, Sarah Klotz, Maria L. Escolar
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Frontiers Media S.A. 2022-07-01
coleção:	Frontiers in Molecular Neuroscience
Assuntos:	Krabbe disease umbilical cord transplantation globoid cell leukodystrophy neurodegeneration peripheral neuropathy
Acesso em linha:	https://www.frontiersin.org/articles/10.3389/fnmol.2022.888231/full
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