首页> 外文OA文献 >Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases

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Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases

机译：从案例报告和临床研究的核心分析结果之间的协议，关于粘性多甲类患者II型（MPS-II）的IDUSULFASE治疗的疗效和安全性。稀有疾病中循证医学的新工具

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Miguel Sampayo-Cordero; Bernat Miguel-Huguet; Almudena Pardo-Mateos; Andrea Malfettone; José Pérez-García; Antonio Llombart-Cussac; Javier Cortés; Marc Moltó-Abad; Cecilia Muñoz-Delgado; Marta Pérez-Quintana; Jordi Pérez-López;
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1. Agreement between the results of meta-analyses from case reports and from clinical studies regarding the efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age: An example of case reports meta-analyses as an useful tool for evidence-based medicine in rare diseases [J] . Sampayo-Cordero Miguel, Miguel-Huguet Bernat, Pardo-Mateos Almudena, Molecular genetics and metabolism . 2018,第2期

机译：从病例报告和临床研究之间的临床研究与临床研究之间的达成协议，关于成人年龄的粘性酶蛋白患者的霉菌酶治疗患者的疗效：案件报告的例子作为证据的有用工具基于罕见疾病的药物
2. The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: “The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis” [J] . Sampayo-Cordero Miguel, Miguel-Huguet Bernat, Malfettone Andrea, Frontiers in Molecular Biosciences . 2021,第a期

机译：不包括罕见疾病的系统评论中的非扫描研究的影响：“荟萃分析的例子评估粘性蛋白患者酶替代治疗的疗效和安全性”
3. Efficacy and safety of intrathecal idursulfase in pediatric patients with mucopolysaccharidosis type II and early cognitive impairment: Design and methods of a controlled, randomized, phase II/III multicenter study [J] . Muenzer Joseph, Burton Barbara K., Harmatz Paul, Molecular genetics and metabolism . 2018,第2期

机译：鞘内耐药病患者II类儿科患者的疗效和安全性和早期认知障碍：受控，随机，II / III多中心研究的设计和方法
4. Is Crowdsourcing Patient-Reported Outcomes the Future of Evidence-Based Medicine? A Case Study of Back Pain [C] . Mor Peleg, Tiffany I. Leung, Manisha Desai, Conference on artificial intelligence in medicine . 2017

机译：病人报告的众包结果是否会超越循证医学的未来？背痛案例研究
5. Mucopolysaccharidosis type IIIB and GalNAc Transferase double knockout mice and additional studies in gene therapy and animal models to assess pathogenesis and therapy. [D] . Mohammed, Eman. 2012

机译：IIIB型粘多糖贮积病和GalNAc转移酶双敲除小鼠以及基因治疗和动物模型的其他研究，以评估发病机理和治疗方法。
6. Agreement between results of meta-analyses from case reports and clinical studies regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases [O] . Miguel Sampayo-Cordero, Bernat Miguel-Huguet, Almudena Pardo-Mateos, 2019

机译：病例报告和临床研究的荟萃分析结果关于艾杜糖酶治疗II型粘多糖贮积病（MPS-II）的有效性和安全性之间的一致性。罕见病中循证医学的新工具
7. The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: “The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis” [O] . Miguel Sampayo-Cordero, Bernat Miguel-Huguet, Andrea Malfettone, 2021

机译：不包括稀有疾病系统评论中的非扫描研究的影响：“荟萃分析的例子评估粘性多甲患者酶替代疗法的疗效和安全性”

Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases

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