View clinical trials related to Myasthenic Syndromes, Congenital.
Filter by:A few recent observational studies show that despite the lack of clear recommendations, many individuals with myasthenia participate in exercise. However, no link has been found between exercise and the severity of myasthenia symptoms, measured by the Muscle Myasthenia Score or the Myasthenia Gravis Composite Score. This suggests that there are other factors that may limit or prevent some individuals from being active, or factors which may facilitate participation in exercise. Studies in other diseases have shown that there are internal (beliefs, motivation, etc.) and external (cost, accessibility, etc.) factors unrelated to the disease which may play a role in exercise participation. The aim of this study is to identify factors that facilitate or limit exercise in individuals with autoimmune myasthenia gravis, congenital myasthenia syndrome and Lambert-Eaton syndrome.
This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 and above) diagnosed with certain genetic subtypes of CMS and demonstrated open label (amifampridine phosphate) or history of sustained amifampridine benefit from treatment.
This is a retrospective study that follows the clinical evolution, the pregnancy and the post-partum perioad of female patients with Congenital Myasthenic Syndrome.
The study tests the notion that patients suffering from certain types of congenital myasthenic syndromes are benefitted by the use of Albuterol at doses used in clinical practice.