Cipaglucosidase alfa and miglustat for treatment of late-onset Pompe disease (LOPD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).
作者信息
Baker Elizabeth K, Derry Allyson, Cohen Jennifer L, Chang Irene J
机构信息
Division of Medical Genetics, Department of Pediatrics, Atrium Health Levine Children's Hospital, Charlotte, NC.
Lewis Katz School of Medicine at Temple University, Philadelphia, PA.
出版信息
Genet Med Open. 2025 Aug 25;3:103444. doi: 10.1016/j.gimo.2025.103444. eCollection 2025.
Abstract
摘要
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本文引用的文献
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Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
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