Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry

被引：0

作者：

Priya S. Kishnani

David Kronn

Shugo Suwazono

Alexander Broomfield

Juan Llerena

Zuhair Nasser Al-Hassnan

Julie L. Batista

Kathryn M. Wilson

Magali Periquet

Nadia Daba

Andreas Hahn

Yin-Hsiu Chien

机构：

[1] Duke University Medical Center,Division of Medical Genetics, Department of Pediatrics

[2] New York Medical College,Department of Pathology and Pediatrics

[3] National Hospital Organization Okinawa National Hospital,Center for Clinical Neuroscience

[4] Central Manchester Foundation Trust,Willink Biochemical Genetics Unit, Manchester Center for Genomic Medicine, St Mary’s Hospital

[5] Instituto Fernandes Figueira/FIOCRUZ,Centro de Genética Médica

[6] King Faisal Specialist Hospital and Research Center,Department of Medical Genomics, Center for Genomic Medicine

[7] Sanofi,Department of Child Neurology

[8] Navitas Data Sciences,Department of Medical Genetics and Pediatrics

[9] Sanofi,undefined

[10] Sanofi,undefined

[11] University Hospital Giessen,undefined

[12] National Taiwan University Hospital,undefined

来源：

Orphanet Journal of Rare Diseases | / 18卷

关键词：

Alglucosidase alfa; Dose; Enzyme replacement therapy; Infantile onset Pompe disease; Pompe disease; Pompe registry;

D O I：

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学科分类号：

摘要：

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