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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Detalhes bibliográficos
Publicado no:JAMA
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: American Medical Association 2018
Assuntos:
Original Investigation
Acesso em linha: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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