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...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	JAMA : the journal of the American Medical Association 2018-12, Vol.320 (22), p.2344-2353
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Bayes Theorem Bayesian analysis Clinical trials Conditional probability Design Diseases Double-Blind Method Drug therapy Feasibility studies Female Genotypes Heterogeneity Humans Hypersensitivity Impact analysis Male Medical treatment Mexiletine - adverse effects Mexiletine - therapeutic use Models, Statistical Muscles Muscular system Myotonia Myotonia - drug therapy Myotonic Disorders - drug therapy Original Investigation Patients Randomized Controlled Trials as Topic Rare Diseases Reduction Skin Sodium channels Stiffness Subgroups Voltage-Gated Sodium Channel Blockers - adverse effects Voltage-Gated Sodium Channel Blockers - therapeutic use
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!