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  1. Article

    Open Access

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

    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 haematopoieti...

    Abhijit Ricky Pal, Eveline J Langereis… in Orphanet Journal of Rare Diseases (2015)

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