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Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for young Hurler patients. Despite halting of neurocognitive decline and improvement of life expectancy, the beneficial effect on the skeletal system is limited. As orthopedic complications are one of the most disa...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Schmidt, Mona, Breyer, Sandra, Löbel, Ulrike, Yarar, Sinef, Stücker, Ralf, Ullrich, Kurt, Müller, Ingo, Muschol, Nicole
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4938899/
https://ncbi.nlm.nih.gov/pubmed/27392569
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-016-0470-7
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