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Artikel 1 Liver fibrosis during clinical ascertainment of glycogen storage disease type III: a need for improved and systematic monitoring
Enthalten in Genetics in medicine 2.7.2019: 1-9
Online Ressource
Artikel 2 Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease
Enthalten in Genetics in medicine 14.5.2019: 1-9
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Artikel 3 Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry
Enthalten in Orphanet journal of rare diseases Bd. 19, 8.3.2024, Nr. 1, date:12.2024: 1-11
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Artikel 4 Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry
Enthalten in Orphanet journal of rare diseases Bd. 18, 6.12.2023, Nr. 1, date:12.2023: 1-13
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Artikel 5 Response to Heiner-Fokkema et al
Enthalten in Genetics in medicine Bd. 22, 13.7.2020, Nr. 11, date:11.2020: 1917-1918
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