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The patient's view on rare disease trial design - a qualitative study.
Orphanet J Rare Dis. 2019 Feb 7;14(1):31. doi: 10.1186/s13023-019-1002-z.
Orphanet J Rare Dis. 2019.
PMID: 30732630
Free PMC article.
The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design.
Gaasterland CMW, Jansen-van der Weide MC, Vroom E, Leeson-Beevers K, Kaatee M, Kaczmarek R, Bartels B, van der Pol WL, Roes KCB, van der Lee JH.
Gaasterland CMW, et al.
Health Policy. 2018 Dec;122(12):1287-1294. doi: 10.1016/j.healthpol.2018.09.011. Epub 2018 Sep 21.
Health Policy. 2018.
PMID: 30274934
Free article.
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Position statement on access to care in rare liver diseases: advancements of the European reference network (ERN) RARE-LIVER.
Bernts LHP, Jones DEJ, Kaatee MM, Lohse AW, Schramm C, Sturm E, Drenth JPH.
Bernts LHP, et al. Among authors: kaatee mm.
Orphanet J Rare Dis. 2019 Jul 8;14(1):169. doi: 10.1186/s13023-019-1152-z.
Orphanet J Rare Dis. 2019.
PMID: 31287000
Free PMC article.
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