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Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach; study rationale and protocol

BACKGROUND: To obtain evidence for the clinical and cost-effectiveness of treatments for patients with rare diseases is a challenge. Non-dystrophic myotonia (NDM) is a group of inherited, rare muscle diseases characterized by muscle stiffness. The reimbursement of mexiletine, the expert opinion drug...

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Detalles Bibliográficos
Autores principales:	Stunnenberg, Bas C, Woertman, Willem, Raaphorst, Joost, Statland, Jeffrey M, Griggs, Robert C, Timmermans, Janneke, Saris, Christiaan G, Schouwenberg, Bas J, Groenewoud, Hans M, Stegeman, Dick F, van Engelen, Baziel G M, Drost, Gea, van der Wilt, Gert Jan
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2015
Materias:	Study Protocol
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4407841/ https://www.ncbi.nlm.nih.gov/pubmed/25880166 http://dx.doi.org/10.1186/s12883-015-0294-4

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