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The patient's view on rare disease trial design - a qualitative study.
Gaasterland CMW, van der Weide MCJ, du Prie-Olthof MJ, Donk M, Kaatee MM, Kaczmarek R, Lavery C, Leeson-Beevers K, O'Neill N, Timmis O, van Nederveen V, Vroom E, van der Lee JH. Gaasterland CMW, et al. Among authors: van der weide mcj, van nederveen v, van der lee jh. 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. Among authors: van der pol wl, van der lee jh. 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.
Statistical analysis of Goal Attainment Scaling endpoints in randomised trials.
Urach S, Gaasterland C, Posch M, Jilma B, Roes K, Rosenkranz G, Van der Lee JH, Ristl R. Urach S, et al. Among authors: van der lee jh. Stat Methods Med Res. 2019 Jun;28(6):1893-1910. doi: 10.1177/0962280218777896. Epub 2018 Jun 19. Stat Methods Med Res. 2019. PMID: 29921167 Free PMC article.
Applicability and added value of novel methods to improve drug development in rare diseases.
Mitroiu M, Oude Rengerink K, Pontes C, Sancho A, Vives R, Pesiou S, Fontanet JM, Torres F, Nikolakopoulos S, Pateras K, Rosenkranz G, Posch M, Urach S, Ristl R, Koch A, Loukia S, van der Lee JH, Roes KCB. Mitroiu M, et al. Among authors: van der lee jh. Orphanet J Rare Dis. 2018 Nov 12;13(1):200. doi: 10.1186/s13023-018-0925-0. Orphanet J Rare Dis. 2018. PMID: 30419965 Free PMC article.
Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure.
Langereis EJ, Borgo A, Crushell E, Harmatz PR, van Hasselt PM, Jones SA, Kelly PM, Lampe C, van der Lee JH, Odent T, Sakkers R, Scarpa M, Schafroth MU, Struijs PA, Valayannopoulos V, White KK, Wijburg FA. Langereis EJ, et al. Among authors: van hasselt pm, van der lee jh. Orphanet J Rare Dis. 2013 Oct 3;8:155. doi: 10.1186/1750-1172-8-155. Orphanet J Rare Dis. 2013. PMID: 24088413 Free PMC article. Review.
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.
de Ru MH, Boelens JJ, Das AM, Jones SA, van der Lee JH, Mahlaoui N, Mengel E, Offringa M, O'Meara A, Parini R, Rovelli A, Sykora KW, Valayannopoulos V, Vellodi A, Wynn RF, Wijburg FA. de Ru MH, et al. Among authors: van der lee jh. Orphanet J Rare Dis. 2011 Aug 10;6:55. doi: 10.1186/1750-1172-6-55. Orphanet J Rare Dis. 2011. PMID: 21831279 Free PMC article.
149 results