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Journal of Inherited Metabolic Disease
Metadata
Title
Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study
Publication date
01-05-2016
Published in
Journal of Inherited Metabolic Disease / Issue 3/2016
Print ISSN: 0141-8955
Electronic ISSN: 1573-2665
DOI
https://doi.org/10.1007/s10545-015-9912-y

Other articles of this Issue 3/2016

Journal of Inherited Metabolic Disease 3/2016 Go to the issue

Original Article

On the phenotypic spectrum of serine biosynthesis defects

Original Article

Unique plasma metabolomic signatures of individuals with inherited disorders of long-chain fatty acid oxidation

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Intracranial arterial abnormalities in patients with late onset Pompe disease (LOPD)

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Social-cognitive functioning and social skills in patients with early treated phenylketonuria: a PKU-COBESO study

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Acute cortical deafness in a child with MELAS syndrome

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Impact of age at onset and newborn screening on outcome in organic acidurias

ACC 2024 Congress Coverage

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