Amifampridine tablets for the treatment of Lambert-Eaton myasthenic syndrome.
Introduction: Lambert-Eaton myasthenic syndrome is an autoimmune disease of the neuromuscular junction characterized by a presynaptic defect of neuromuscular transmission resulting in muscle weakness and fatigability. Diagnostic features are specific neurophysiological alterations and autoantibody detection. The present review is focused on the use of Amifampridine Phosphate to treat LEMS patients.Areas covered: Medline search from 1990 to 2019 was examined using the free subject terms: Lambert-Eaton myasthenic syndrome, LEMS, Amifampridine, 3,4-diaminopyridine, which were then combined with Treatment, Therapy, Clinical Trial, Controlled Clinical Trial, Randomized Clinical Trial and Cochrane Review. The author has done a supervised analysis of the retrieved articles and focused on those subjectively evaluated as most relevant.Expert commentary: Data from randomized clinical trials and case series have demonstrated that Lambert-Eaton myasthenic syndrome symptoms were successfully treated by Amifampridine Phosphate. Hence, the drug represents a substantial step forward in the symptomatic treatment of the disease due to its efficacy, safety and reliable GMP formulation. As Amifampridine Phosphate works by enhancing the release of acetylcholine at the neuromuscular junction by blocking K+ efflux at the pre-synaptic membrane, it is also conceivable to use it for other diseases of the neuromuscular junction in which such an effect is searched for.
['Mantegazza|Renato|R|https://orcid.org/0000-0002-9810-5737']
D000077770:Amifampridine D006801:Humans D015624:Lambert-Eaton Myasthenic Syndrome D009465:Neuromuscular Agents D026902:Potassium Channel Blockers D016032:Randomized Controlled Trials as Topic D013607:Tablets
Amifampridine Lambert-Eaton myasthenic syndrome neuromuscular junction autoantibody randomized clinical trial 34-diaminopyridine epidemiology