Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia. - Mex vs. placebo in NDMs Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierar ...
Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia. - Mex vs. placebo in NDMs Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierar ...
Patients with non-dystrophic myotonic syndromes (NDMs). NDMs are caused by mutations in the gene encoding for the skeletal muscle sodium (SCN4A) or chloride (CLCN1) channel. In this study patients with NDMs with a genetically confirmed mutation in the SCN4A-gene or the CLCN1-gene as registred in the Netherlands database of NDMs will be included. Patients with non-dystrophic myotonic syndromes (NDMs). NDMs are caused by mutations in the gene enc ...
Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - ND
Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - ND
Myotonic disorders are divided into dystrophic myotonias (DM1 and DM2) and non-dystrophic myotonias (NDM). The dystrophic myotonias are associated with significant progressive muscular weakness and other systemic organ involvement. On the other hand, NDM usually presents with muscle stiffness as the primary symptom, and severe weakness is not considered a major feature, especially in myotonia congenita. MedDRA version: 14.0;Level: LLT;Classification code 10032487;Term: Other specific muscle disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Myotonic disorders are divided into dystrophic myotonias (DM1 and DM2) and non-dystrophic myotonias ...