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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:BMC Neurol
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: BioMed Central 2015
Gaiak:
Study Protocol
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC4407841/
https://ncbi.nlm.nih.gov/pubmed/25880166
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s12883-015-0294-4
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