Fabry disease in children: agalsidase-beta enzyme replacement therapy
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Published in Clinical genetics (01.05.2013)
Published in Clinical genetics (01.05.2013)
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Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study
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Published in Orphanet journal of rare diseases (28.05.2015)
Published in Orphanet journal of rare diseases (28.05.2015)
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