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

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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;
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