Literature DB >> 31486113

Rare disease specialists and clinical pharmacologists unite: Increase collection of longitudinal data!

Gijs W E Santen1, Adam F Cohen2.   

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Year:  2019        PMID： 31486113      PMCID： PMC6848956          DOI： 10.1111/bcp.14079

Source DB:  PubMed          Journal:  Br J Clin Pharmacol        ISSN： 0306-5251            Impact factor:   4.335


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  8 in total

1.  Effect of Mexiletine on Muscle Stiffness in Patients With Nondystrophic Myotonia Evaluated Using Aggregated N-of-1 Trials.

Authors:  Bas C Stunnenberg; Joost Raaphorst; Hans M Groenewoud; Jeffrey M Statland; Robert C Griggs; Willem Woertman; Dick F Stegeman; Janneke Timmermans; Jaya Trivedi; Emma Matthews; Christiaan G J Saris; Bas J Schouwenberg; Gea Drost; Baziel G M van Engelen; Gert Jan van der Wilt
Journal:  JAMA       Date:  2018-12-11       Impact factor: 56.272

Review 2.  Alternative designs for clinical trials in rare diseases.

Authors:  Lusine Abrahamyan; Brian M Feldman; George Tomlinson; Marie E Faughnan; Sindhu R Johnson; Ivan R Diamond; Samir Gupta
Journal:  Am J Med Genet C Semin Med Genet       Date:  2016-11-14       Impact factor: 3.908

Review 3.  The use of biomarkers in human pharmacology (Phase I) studies.

Authors:  A F Cohen; J Burggraaf; J M A van Gerven; M Moerland; G J Groeneveld
Journal:  Annu Rev Pharmacol Toxicol       Date:  2014-10-06       Impact factor: 13.820

Review 4.  Challenges of developing and conducting clinical trials in rare disorders.

Authors:  Lucas Kempf; Jonathan C Goldsmith; Robert Temple
Journal:  Am J Med Genet A       Date:  2017-08-16       Impact factor: 2.802

5.  Targeted therapy in patients with PIK3CA-related overgrowth syndrome.

Authors:  Quitterie Venot; Thomas Blanc; Smail Hadj Rabia; Laureline Berteloot; Sophia Ladraa; Jean-Paul Duong; Estelle Blanc; Simon C Johnson; Clément Hoguin; Olivia Boccara; Sabine Sarnacki; Nathalie Boddaert; Stephanie Pannier; Frank Martinez; Sato Magassa; Junna Yamaguchi; Bertrand Knebelmann; Pierre Merville; Nicolas Grenier; Dominique Joly; Valérie Cormier-Daire; Caroline Michot; Christine Bole-Feysot; Arnaud Picard; Véronique Soupre; Stanislas Lyonnet; Jeremy Sadoine; Lotfi Slimani; Catherine Chaussain; Cécile Laroche-Raynaud; Laurent Guibaud; Christine Broissand; Jeanne Amiel; Christophe Legendre; Fabiola Terzi; Guillaume Canaud
Journal:  Nature       Date:  2018-06-13       Impact factor: 49.962

6.  Enhancing crossover trial design for rare diseases: limiting ineffective exposure and increasing study power by enabling patient choice to escape early.

Authors:  Bin Huang; Edward H Giannini; Daniel J Lovell; Lili Ding; Yongchao Liu; Philip J Hashkes
Journal:  Contemp Clin Trials       Date:  2014-05-12       Impact factor: 2.226

Review 7.  Patient reported outcome measures in rare diseases: a narrative review.

Authors:  Anita Slade; Fatima Isa; Derek Kyte; Tanya Pankhurst; Larissa Kerecuk; James Ferguson; Graham Lipkin; Melanie Calvert
Journal:  Orphanet J Rare Dis       Date:  2018-04-23       Impact factor: 4.123

Review 8.  Clinical and translational pharmacological aspects of the management of fibrous dysplasia of bone.

Authors:  Marlous Rotman; Neveen Agnes Therese Hamdy; Natasha M Appelman-Dijkstra
Journal:  Br J Clin Pharmacol       Date:  2018-12-25       Impact factor: 4.335
  8 in total

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