Author:
Muenzer Joseph,Jones Simon A.,Tylki-Szymańska Anna,Harmatz Paul,Mendelsohn Nancy J.,Guffon Nathalie,Giugliani Roberto,Burton Barbara K.,Scarpa Maurizio,Beck Michael,Jangelind Yvonne,Hernberg-Stahl Elizabeth,Larsen Maria Paabøl,Pulles Tom,Whiteman David A. H.
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Genetics(clinical),General Medicine
Reference45 articles.
1. Potter BK, Khangura SD, Tingley K, Chakraborty P, Little J. Translating rare-disease therapies into improved care for patients and families: what are the right outcomes, designs, and engagement approaches in health-systems research? Genet Med. 2016;18(2):117–23.
2. Jones S, James E, Prasad S. Disease registries and outcomes research in children: focus on lysosomal storage disorders. Paediatr Drugs. 2011;13(1):33–47.
3. Registries for Evaluating Patient Outcomes: A User's Guide. In: Gliklich RE, Dreyer NA, Leavy MB, editors. Registries for Evaluating Patient Outcomes: A User's Guide. 3rd ed. Rockville: Agency for Healthcare Research and Quality (US); 2014.
4. Rare Disease Registries in Europe. Orphanet Report Series, Rare Diseases collection [ http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf ]. Accessed 23 Apr 2017.
5. Byrne BJ, Kishnani PS, Case LE, Merlini L, Muller-Felber W, Prasad S, van der Ploeg A. Pompe disease: design, methodology, and early findings from the Pompe Registry. Mol Genet Metab. 2011;103(1):1–11.
Cited by
48 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