Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome

BACKGROUND: The lysosomal storage disorder, mucopolysaccharidosis I (MPS I), commonly manifests with upper airway obstruction and sleep disordered breathing (SDB). The success of current therapies, including haematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT) may be...

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Bibliografiske detaljer
Udgivet i:Orphanet J Rare Dis
Main Authors: Pal, Abhijit Ricky, Langereis, Eveline J, Saif, Muhammad A, Mercer, Jean, Church, Heather J, Tylee, Karen L, Wynn, Robert F, Wijburg, Frits A, Jones, Simon A, Bruce, Iain A, Bigger, Brian W
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2015
Fag:
Research
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4450482/
https://ncbi.nlm.nih.gov/pubmed/25887468
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0255-4
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