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Journal of Neurology|June 12, 2020
Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment startDavid W Stockton, Priya Kishnani, Ans van der Ploeg, et al.
Pharmacoepidemiology and Drug Safety|December 16, 2011
Source document verification in the Mucopolysaccharidosis Type I RegistryKarien Verhulst, Laura Artiles-Carloni, Michael Beck, et al.
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