Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.,Genetics in Medicine

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Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
Genetics in Medicine ( IF 8.8 ) Pub Date : 2018-10-01 , DOI: 10.1038/gim.2018.2
Si Houn Hahn ₁ , David Kronn ₂ , Nancy D Leslie ₃ , Loren D M Pena ₄ , Pranoot Tanpaiboon ₅ , Michael J Gambello ₆ , James B Gibson ₇ , Richard Hillman ₈ , David W Stockton ₉ , John W Day ₁₀ , Raymond Y Wang _{11,

12} , Kristina An Haack ₁₃ , Raheel Shafi ₁₃ , Susan Sparks ₁₃ , Yang Zhao ₁₃ , Catherine Wilson ₁₃ , Priya S Kishnani ₄ ,

Affiliation

Departments of Pediatrics and Medicine and Biochemical Genetics Program, Seattle Children's Hospital/University of Washington, Seattle, Washington, USA.
Pediatrics, New York Medical College, Valhalla, New York, USA.
Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Pediatrics, Duke University Medical Center, Durham, North Carolina, USA.
Genetics, Children's National Health System, Washington, DC, USA.
Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA.
Clinical and Metabolic Genetics, Dell Children's Medical Group, Austin, Texas, USA.
Professor Emeritus, University of Missouri Child Health, Columbia, Missouri, USA.
Division of Genetic, Genomic, and Metabolic Disorders, Wayne State University and Children's Hospital of Michigan, Detroit, Michigan, USA.
Neurology, Pediatrics, and Medical Genetics, Stanford University, Stanford, California, USA.
Lysosomal Storage Disease Program, Children's Hospital of Orange County, Orange, California, USA.
Pediatrics, University of California-Irvine School of Medicine, Orange, California, USA.
Sanofi Genzyme, Cambridge, Massachusetts, USA.

Pompe disease results from lysosomal acid α-glucosidase (GAA) deficiency and its associated glycogen accumulation and muscle damage. Alglucosidase alfa (recombinant human GAA (rhGAA)) received approval in 2006 as a treatment for Pompe disease at the 160 L production scale. In 2010, larger-scale rhGAA was approved for patients up to 8 years old without cardiomyopathy. NCT01526785 evaluated 4,000 L rhGAA efficacy/safety in US infantile- or late-onset Pompe disease (IOPD, LOPD) patients up to 1 year old transitioned from 160 L rhGAA.

中文翻译：

在美国庞贝病儿童和青少年中以 4,000 升规模生产的阿糖苷酶 α 的功效、安全性和免疫原性：ADVANCE，一项 IV 期、开放标签、前瞻性研究。

庞贝病是由溶酶体酸 α-葡萄糖苷酶 (GAA) 缺乏及其相关的糖原积累和肌肉损伤引起的。阿糖苷酶 α（重组人 GAA (rhGAA)）于 2006 年获得批准，可用于 160 L 生产规模的庞贝病治疗。2010 年，更大规模的 rhGAA 被批准用于 8 岁以下无心肌病的患者。NCT01526785 评估了 4,000 L rhGAA 在从 160 L rhGAA 过渡到 1 岁以下的美国婴儿或迟发性庞贝病 (IOPD, LOPD) 患者中的疗效/安全性。

更新日期：2018-03-23

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