Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act
Author:
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Genetics (clinical),General Medicine
Link
http://link.springer.com/content/pdf/10.1186/s13023-015-0262-5.pdf
Reference37 articles.
1. Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA. 1999;281(3):249–54.
2. Poorthuis BJ, Wevers RA, Kleijer WJ, Groener JE, de Jong JG, van Weely S, et al. The frequency of lysosomal storage diseases in The Netherlands. Hum Genet. 1999;105(1–2):151–6.
3. Applegarth DA, Toone JR, Lowry RB. Incidence of inborn errors of metabolism in British Columbia, 1969–1996. Pediatrics. 2000;105(1), e10.
4. Pinto R, Caseiro C, Lemos M, Lopes L, Fontes A, Ribeiro H, et al. Prevalence of lysosomal storage diseases in Portugal. Eur J Hum Genet. 2004;12(2):87–92. doi:10.1038/sj.ejhg.5201044.
5. Platt F, Walkley S. Lysosomal Disorders of the Brain: Recent Advances in Molecular and Cellular Pathogenesis and Treatment. Oxford: Oxford University Press; 2004.
Cited by 41 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
1. Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders;Advanced Drug Delivery Reviews;2023-06
2. Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies;American Journal of Medical Genetics Part C: Seminars in Medical Genetics;2023-02-04
3. Bringing enzymes to the proximity party;RSC Chemical Biology;2023
4. Orphan drug development in alpha-1 antitypsin deficiency;Scientific Reports;2022-09-15
5. The past, present, and future of enzyme-based therapies;Drug Discovery Today;2022-01
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3