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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Dades bibliogràfiques
Publicat a:JAMA
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: American Medical Association 2018
Matèries:
Original Investigation
Accés en línia: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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