Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry

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.

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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.

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