title: 3,4-diaminopyridine in childhood myasthenia: double-blind, placebo-controlled trial.
abstract: Eleven patients with congenital and five with juvenile myasthenia gravis, aged 5 to 24 years, were given 3,4-diaminopyridine in a double-blind, placebo-controlled, crossover study. Clinical improvement was observed in 5 of 11 congenital myasthenia patients, and placebo effect, in 3 of 11. Juvenile myasthenia patients did not respond. Single-fiber electromyographic studies did not reveal any changes correlating with the clinical status of the patient. This study demonstrates the importance of double-blind and placebo-controlled studies to determine the effect of 3,4-diaminopyridine in congenital myasthenia. This drug may have different effects on various presynaptic and postsynaptic defects of neuromuscular transmission resulting in congenital myasthenia syndromes.
authors.affiliation: Department of Pediatric Neurology, Hacettepe University, Ankara, Turkey.
authors.forename: B
authors.initials: B
authors.lastname: Anlar
authors.forename: K
authors.initials: K
authors.lastname: Varli
authors.forename: E
authors.initials: E
authors.lastname: Ozdirim
authors.forename: M
authors.initials: M
authors.lastname: Ertan
mesh_terms: D015761:4-Aminopyridine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D000293:Adolescent; D000328:Adult; D000077770:Amifampridine; D002648:Child; D002675:Child, Preschool; D018592:Cross-Over Studies; D004311:Double-Blind Method; D004576:Electromyography / Q000187:drug effects; D005260:Female; D006801:Humans; D008297:Male; D009157:Myasthenia Gravis / Q000151:congenital / Q000175:diagnosis / Q000188:drug therapy*; D009460:Neurologic Examination / Q000187:drug effects
chemical_list: D015761:4-Aminopyridine; D000077770:Amifampridine