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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:JAMA
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Medical Association 2018
Gaiak:
Original Investigation
Sarrera elektronikoa: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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