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Journal of Child Neurology
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3,4-Diaminopyridine in Childhood Myasthenia: Double-Blind, Placebo-Controlled Trial

Banu Anlar, MD

Department of Pediatric Neurology, Hacettepe University, Ankara, Turkey

Kubilay Varli, MD

Department of Neurology, Hacettepe University, Ankara, Turkey

Emire Ozdirim, MD

Department of Pediatric Neurology, Hacettepe University, Ankara, Turkey

Mevlut Ertan, PhD

Faculty of Pharmacy Hacettepe University, Ankara, Turkey

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. (J Child Neurol 1996;11:458-461).

Journal of Child Neurology, Vol. 11, No. 6, 458-461 (1996)
DOI: 10.1177/088307389601100608
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D. B. Sanders, J. M. Massey, L. L. Sanders, and L. J. Edwards
A randomized trial of 3,4-diaminopyridine in Lambert-Eaton myasthenic syndrome
Neurology, February 8, 2000; 54(3): 603 - 603.
[Abstract] [Full Text] [PDF]