The evolution of pulmonary function in childhood onset Mucopolysaccharidosis type I

Respiratory outcomes in Mucopolysaccharidosis Type I (MPS I), have mainly focused on upper airway obstruction, with the evolution of the restrictive lung disease being poorly documented. We report the long-term pulmonary function outcomes and examine the potential factors affecting these in 2 cohort...

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Veröffentlicht in:	Molecular genetics and metabolism 2021-02, Vol.132 (2), p.94-99
Hauptverfasser:	Broomfield, A., Sims, J., Mercer, J., Hensman, P., Ghosh, A., Tylee, K., Stepien, K.M., Oldham, A., Prathivadi Bhayankaram, N., Wynn, R., Wright, N.B., Jones, S.A., Wilkinson, S.
Format:	Artikel
Sprache:	eng
Schlagworte:	Enzyme replacement Haemopoietic stem cell transplantation Hurler Mucopolysaccharidosis type I Pulmonary function Restrictive lung disease
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