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: B Anlar, K Varli, E Ozdirim, M Ertan
• Journal: Journal of child neurology (J Child Neurol) Vol. 11 Issue 6 Pg. 458-61 (Nov 1996) ISSN: 0883-0738 [Print] United States
• PMID: 9120223 (Publication Type: Clinical Trial, Journal Article, Randomized Controlled Trial)

Chemical References

• 4-Aminopyridine
• Amifampridine

Topics

• 4-Aminopyridine (analogs & derivatives, therapeutic use)
• Adolescent
• Adult
• Amifampridine
• Child
• Child, Preschool
• Cross-Over Studies
• Double-Blind Method
• Electromyography (drug effects)
• Female
• Humans
• Male
• Myasthenia Gravis (congenital, diagnosis, drug therapy)
• Neurologic Examination (drug effects)