Author:
Bendixen Roxanna M.,Morgenroth Lauren P.,Clinard Kristin L.
Subject
Pharmacology (medical),Pharmacology
Reference39 articles.
1. What the orphan drug act has done lately for children with rare diseases: a 10-year analysis;Thorat;Pediatrics,2012
2. Vickers PJ. Challenges and opportunities in the treatment of rare diseases. Drug Discovery World, spring 2013. Retrieved from: http://www.ddw-online.com/therapeutics/p211490-challenges-and-opportunities-in-the-treatment-of-rare-diseases-spring-13.html. Accessed on January 22, 2016.
3. The TREAT-NMD Duchenne muscular dystrophy registries: conceptions, design and utilization by industry and academia;Bladen;Human Mut,2013
4. Collaborating to bring new therapies to the patient—the TREAT-NMD model;Bushby;Acta Myologica,2009
5. Practical aspects of recruitment and retention in clinical trials of rare genetic diseases: the phenylketonuria (PKU) experience;DeWard;J Genet Counseling,2014
Cited by
26 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