罕见疾病及孤儿药物研究现状及进展
Current views on rare diseases research and orphan drugs development
投稿时间:2011-02-17  
DOI:  
中文关键词:罕见疾病,孤儿药物,治疗,研发
英文关键词:rare diseases, orphan drugs, therapy, research and development
基金项目:
作者单位E-mail
江静雯 中国科学技术大学生命科学学院合肥 230027中国科学院微生物研究所 中国科学院病原微生物与免疫学重点实验室北京 100101  
李晶 中国科学院微生物研究所 中国科学院病原微生物与免疫学重点实验室北京 100101  
刘文军 中国科学院微生物研究所 中国科学院病原微生物与免疫学重点实验室北京 100101 liuwj@im.ac.cn 
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中文摘要:
      文中简述了罕见疾病的定义、发病原因、分类,总结了国内外罕见疾病研究和孤儿药物研发的现状,分析了生物技术的研究手段在罕见疾病药物研发方面的应用,进一步阐述了罕见疾病研究和孤儿药物开发的必要性和紧迫性,对推动科学技术的进步和人类健康事业的发展具有深远的意义。
英文摘要:
      Interest in rare diseases research and orphan drugs development has been increased distinctly in recent years. The number of affected people with rare diseases is considerable around the world and the formulation of national and international incentive policies to accelerate orphan drugs development, aiming at offering facilities and necessary conditions for patient access to treatment, gains favorable results. In particular, more measures should be taken to catalyze further progress due to behindhand level in this field in China. Additionally, therapeutic methods of rare diseases were also discussed.
江静雯,李晶,刘文军.罕见疾病及孤儿药物研究现状及进展[J].生物工程学报,2011,27(5):724~729
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