A 10-year study documenting the long-term effectiveness of agalsidase-beta treatment in 52 adult patients with classic Fabry disease

Germain Dominique P.; Charrow Joel; Desnick Robert J.; Guffon Nathalie; Kempf Judy; Lachmann Robin H.; Lemay Roberta; Linthorst Gabor E.; Packman Seymour; Scott C. Ronald; Waldek Stephen; Warnock David G.; Weinreb Neal J.; Wilcox William R.

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A 10-year study documenting the long-term effectiveness of agalsidase-beta treatment in 52 adult patients with classic Fabry disease

机译：一项为期10年的研究记录了Agalsidase-beta治疗52例经典Fabry病成年患者的长期有效性

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《Molecular genetics and metabolism 》 |2015年第2期| 共2页
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Germain Dominique P.; Charrow Joel; Desnick Robert J.; Guffon Nathalie; Kempf Judy; Lachmann Robin H.; Lemay Roberta; Linthorst Gabor E.; Packman Seymour; Scott C. Ronald; Waldek Stephen; Warnock David G.; Weinreb Neal J.; Wilcox William R.;
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中图分类内分泌腺疾病及代谢病 ;
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1. A 10-year study documenting the long-term effectiveness of agalsidase-beta treatment in 52 adult patients with classic Fabry disease [J] . Germain Dominique P., Charrow Joel, Desnick Robert J., Molecular genetics and metabolism . 2015 ,第2期

机译：一项为期10年的研究记录了Agalsidase-beta治疗52例经典Fabry病成年患者的长期有效性
2. Effect of long-term migalastat treatment on plasma globotriaosylsphingosine (lyso-Gb(3)) levels in patients with Fabry disease previously treated with enzyme replacement therapy: Results from ATTRACT and open-label extension studies [J] . Bichet Daniel, Barth Jay A., Williams Hadis, Molecular genetics and metabolism . 2019 ,第2期

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3. Long-Term Effects of Rivastigmine Treatment on the Need for Psychotropic Medications in Nursing Home Patients with Alzheimer's Disease Results of a 52-Week Open-Label Study [J] . Keith Edwards, Barbara Koumaras, Michael Chen, Clinical drug investigation . 2005 ,第8期

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4. New Anti-Microbial Treatment of Purulent-Inflammatory Lung Diseases in Patients Supported by Long-Term Artificial Ventilation of Lungs [C] . EG. Nazirov, R.A. Ibadov, Z.A. Shanieva, NATO Advanced Study Institute on Biodefence: Advanced materials and methods for health protection . 2011

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5. Patients with advanced heart failure: A description of the disease trajectory in the study to understand prognoses and preferences for outcomes and risks of treatment in critically ill adults (SUPPORT) [D] . Castle, Jane Elizabeth 2000

机译：晚期心力衰竭患者：描述研究中的疾病轨迹，以了解重症成人的预后和对结局和治疗风险的偏好（支持）
6. Agalsidase beta treatment slows estimated glomerular filtration rate loss in classic Fabry disease patients: results from an individual patient data meta-analysis [O] . Alberto Ortiz, Steve Kanters, Alaa Hamed, 2021

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A 10-year study documenting the long-term effectiveness of agalsidase-beta treatment in 52 adult patients with classic Fabry disease

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