Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act

机译：罕见风湿病的新型治疗方法：美国孤儿药法案30年的影响分析

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BackgroundRare rheumatologic diseases are a heterogeneous group of conditions associated with high morbidity. As a whole group, rare rheumatologic diseases afflict millions of people demanding for effective therapies. Therefore, we analyzed the impact of the US Orphan Drug Act on the development of anti-rheumatic orphan drugs.

机译：背景罕见的风湿病是一组与高发病率相关的异质性疾病。总体而言，罕见的风湿病困扰着数百万需要有效治疗的人。因此，我们分析了《美国孤儿药法》对抗风湿性孤儿药发展的影响。

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期刊名称 Orphanet Journal of Rare Diseases
作者
Thomas Lutz; Anette Lampert; Georg F. Hoffmann; Markus Ries;
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作者单位

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年(卷),期 2016(11),-1
年度 2016
页码 60
总页数 12
原文格式 PDF
正文语种
中图分类医学史;
关键词
Orphan drug act Orphan drug development Rare disease Rare rheumatologic disease;

机译：孤儿药法案;孤儿药开发;罕见病;罕见风湿病;

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专利

1. Novel orphan medicines and abandoned pathways - the US Orphan Drug Act of 1983 and the impact on rare rheumatologic diseases and lysosomal storage disorders [J] . Markus Ries, Thomas Lutz, Anette Lampert, Molecular and Cellular Pediatrics . 2015,第S1期

机译：新型的孤儿药和被遗弃的途径-1983年的美国孤儿药法及其对罕见的风湿病和溶酶体贮积病的影响
2. A 30-year retrospective: National Organization for Rare Disorders, the Orphan Drug Act, and the role of rare disease patient advocacy groups [J] . Mary Dunkle Orphan Drugs: Research and Reviews . 2014,第2期

机译：30年回顾展：国家罕见病组织，《孤儿药法》以及罕见病患者倡导团体的作用
3. Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act [J] . Konstantin Mechler, William K Mountford, Georg F Hoffmann, Orphanet journal of rare diseases . 2015,第S1期

机译：溶酶体贮积病中药物开发的压力–距离美国孤儿药法案三十年的定量分析
4. Spatial Analysis of Drug Poisoning Deaths and Access to Substance-use Disorder Treatment in the United States [C] . Yelena Ogneva-Himmelberger International Conference on Geographical Information Systems Theory, Applications and Management . 2019

机译：美国药物中毒死亡的空间分析及对美国物质使用障碍治疗
5. Meta-Analysis to Identify and Evaluate Factors Associated with Regulatory Approval of Orphan Drugs (OD) to Develop an Algorithm for Predicting Regulatory Approval (Success) and to Develop a Standardized Tool to Improve Orphan Drug Portfolio Decision-Making [D] . Florent, Milky C. 2019

机译：荟萃分析，以识别和评估与孤儿药物监管批准（OD）相关的因素，以开发预测监管批准（成功）的算法，并开发标准化工具以改善孤儿药物投资组合决策
6. Novel orphan medicines and abandoned pathways - the US Orphan Drug Act of 1983 and the impact on rare rheumatologic diseases and lysosomal storage disorders [O] . Markus Ries, Thomas Lutz, Anette Lampert, 2015

机译：新型的孤儿药和被遗弃的途径-1983年的美国孤儿药法案及其对罕见的风湿病和溶酶体贮积病的影响
7. Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act [O] . Lutz, Thomas, Lampert, Anette, Hoffmann, Georg F., 2016

机译：罕见风湿病的新型治疗方法：美国孤儿药法案30年的影响分析

Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act

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