Effect of Mexiletine on Muscle Stiffness in Patients With Nondystrophic Myotonia Evaluated Using Aggregated N-of-1 Trials

Groft, 2013, Rare diseases research: expanding collaborative translational research opportunities., Chest, 144, 16, 10.1378/chest.13-0606

Haffner, 2006, Adopting orphan drugs—two dozen years of treating rare diseases., N Engl J Med, 354, 445, 10.1056/NEJMp058317

Augustine, 2013, Clinical trials in rare disease: challenges and opportunities., J Child Neurol, 28, 1142, 10.1177/0883073813495959

Guyatt, 1986, Determining optimal therapy—randomized trials in individual patients., N Engl J Med, 314, 889, 10.1056/NEJM198604033141406

Zucker, 1997, Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment., J Clin Epidemiol, 50, 401, 10.1016/S0895-4356(96)00429-5

Trivedi, 2013, Non-dystrophic myotonia: prospective study of objective and patient reported outcomes., Brain, 136, 2189, 10.1093/brain/awt133

Statland, 2012, Mexiletine for symptoms and signs of myotonia in nondystrophic myotonia: a randomized controlled trial., JAMA, 308, 1357, 10.1001/jama.2012.12607

Stunnenberg, 2015, Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach: study rationale and protocol., BMC Neurol, 15, 43, 10.1186/s12883-015-0294-4

van Engelen, 2007, The Dutch neuromuscular database CRAMP (Computer Registry of All Myopathies and Polyneuropathies): development and preliminary data., Neuromuscul Disord, 17, 33, 10.1016/j.nmd.2006.09.017

Statland, 2011, An interactive voice response diary for patients with non-dystrophic myotonia., Muscle Nerve, 44, 30, 10.1002/mus.v44.1

Sansone, 2012, Measuring quality of life impairment in skeletal muscle channelopathies., Eur J Neurol, 19, 1470, 10.1111/j.1468-1331.2012.03751.x

Seesing, 2014, Shared medical appointments improve QOL in neuromuscular patients: a randomized controlled trial., Neurology, 83, 240, 10.1212/WNL.0000000000000588

Aaronson, 1998, Translation, validation, and norming of the Dutch language version of the SF-36 Health Survey in community and chronic disease populations., J Clin Epidemiol, 51, 1055, 10.1016/S0895-4356(98)00097-3

Trip, 2009, Redefining the clinical phenotypes of non-dystrophic myotonic syndromes., J Neurol Neurosurg Psychiatry, 80, 647, 10.1136/jnnp.2008.162396

Podsiadlo, 1991, The timed “Up & Go”: a test of basic functional mobility for frail elderly persons., J Am Geriatr Soc, 39, 142, 10.1111/jgs.1991.39.issue-2

Hammarén, 2005, Quantification of mobility impairment and self-assessment of stiffness in patients with myotonia congenita by the physiotherapist., Neuromuscul Disord, 15, 610, 10.1016/j.nmd.2005.07.002

Statland, 2012, A quantitative measure of handgrip myotonia in non-dystrophic myotonia., Muscle Nerve, 46, 482, 10.1002/mus.v46.4

Streib, 1987, AAEE minimonograph #27: differential diagnosis of myotonic syndromes., Muscle Nerve, 10, 603, 10.1002/(ISSN)1097-4598

Li, 2007, Simultaneous determination of ten antiarrhythic drugs and a metabolite in human plasma by liquid chromatography—tandem mass spectrometry., J Chromatogr B Analyt Technol Biomed Life Sci, 847, 174, 10.1016/j.jchromb.2006.10.013

Huber, 2007, Amitriptyline to relieve pain in juvenile idiopathic arthritis: a pilot study using Bayesian metaanalysis of multiple N-of-1 clinical trials., J Rheumatol, 34, 1125

Mitchell, 2015, The effect of methylphenidate on fatigue in advanced cancer: an aggregated N-of-1 trial., J Pain Symptom Manage, 50, 289, 10.1016/j.jpainsymman.2015.03.009

Nathan, 2006, A pilot study of ondansetron plus metopimazine vs. ondansetron monotherapy in children receiving highly emetogenic chemotherapy: a Bayesian randomized serial N-of-1 trials design., Support Care Cancer, 14, 268, 10.1007/s00520-005-0875-7

Nikles, 2017, A series of N-of-1 trials of stimulants in brain injured children., NeuroRehabilitation, 40, 11, 10.3233/NRE-161386

Sung, 2007, Serial controlled N-of-1 trials of topical vitamin E as prophylaxis for chemotherapy-induced oral mucositis in paediatric patients., Eur J Cancer, 43, 1269, 10.1016/j.ejca.2007.02.001

Logigian, 2010, Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1., Neurology, 74, 1441, 10.1212/WNL.0b013e3181dc1a3a

Suetterlin, 2015, Long-term safety and efficacy of mexiletine for patients with skeletal muscle channelopathies., JAMA Neurol, 72, 1531, 10.1001/jamaneurol.2015.2338

Spiegelhalter, 2004, Bayesian Approaches to Clinical Trials and Health-Care Evaluation

JAMA Guide to Statistics and Methods. JAMA Network website. https://jamanetwork.com/collections/44042/guide-to-statistics-and-medicine. Accessed October 29, 2018.