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News From the Food and Drug Administration
June 18, 2019

First Treatment for Children With Rare Myasthenic Syndrome

JAMA. 2019;321(23):2274. doi:10.1001/jama.2019.7979

Amifampridine tablets have received approval for treating children aged 6 to 17 years who have Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disease that causes debilitating muscle weakness.

“This approval will provide a much-needed treatment option for pediatric patients with LEMS who have significant weakness and fatigue that can often cause great difficulties with daily activities,” Billy Dunn, MD, director of the FDA’s Division of Neurology Products, said in a statement.