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

IMPORTANCE: In rare diseases it is difficult to achieve high-quality evidence of treatment efficacy because of small cohorts and clinical heterogeneity. With emerging treatments for rare diseases, innovative trial designs are needed. OBJECTIVE: To investigate the effectiveness of mexiletine in nondy...

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Опубликовано в: :JAMA
Главные авторы: Stunnenberg, Bas C., Raaphorst, Joost, Groenewoud, Hans M., Statland, Jeffrey M., Griggs, Robert C., Woertman, Willem, Stegeman, Dick F., Timmermans, Janneke, Trivedi, Jaya, Matthews, Emma, Saris, Christiaan G. J., Schouwenberg, Bas J., Drost, Gea, van Engelen, Baziel G. M., van der Wilt, Gert Jan
Формат: Artigo
Язык:Inglês
Опубликовано: American Medical Association 2018
Предметы:
Original Investigation
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6583079/
https://ncbi.nlm.nih.gov/pubmed/30535218
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jama.2018.18020
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