title: 3,4-Diaminopyridine in the treatment of congenital (hereditary) myasthenia.
abstract: Congenital or hereditary myasthenia describes a heterogeneous group of disorders in which the immune system is not implicated. Treatment has previously depended on anticholinesterase medication. The effectiveness of 3,4-diaminopyridine (3,4-DAP), a preparation that enhances acetylcholine release from motor nerve terminals, has been evaluated using a series of standardised strength measures. Sixteen patients (aged seven to 47 years) were studied in an open prospective trial, and four of them in a double blind crossover trial; existing anticholinesterase medication was continued. For the group as a whole, there was a highly significant increase in muscle strength (p less than 0.001; n = 16). In individual paired comparisons, 13 out of 16 showed significant improvement in the open trial and four out of four in the blind crossover trial. In conclusion, 3,4-DAP, either alone or combined with anticholinesterase medication, may be a useful additional treatment in congenital myasthenia.
authors.affiliation: Department of Neurology, Radcliffe Infirmary, University of Oxford, UK.
authors.forename: J
authors.initials: J
authors.lastname: Palace
authors.forename: C M
authors.initials: CM
authors.lastname: Wiles
authors.forename: J
authors.initials: J
authors.lastname: Newsom-Davis
mesh_terms: D015761:4-Aminopyridine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D000293:Adolescent; D000328:Adult; D000077770:Amifampridine; D002648:Child; D004576:Electromyography; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009132:Muscles / Q000503:physiopathology; D009468:Neuromuscular Diseases / Q000151:congenital / Q000188:drug therapy* / Q000503:physiopathology; D011446:Prospective Studies; D013997:Time Factors
chemical_list: D015761:4-Aminopyridine; D000077770:Amifampridine