Engaging Participants in Rare Disease Research: A Qualitative Study of Duchenne Muscular Dystrophy

Author:

Bendixen Roxanna M.,Morgenroth Lauren P.,Clinard Kristin L.

Publisher

Elsevier BV

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

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