Patients with a rare form of epilepsy now have a new treatment option

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Children's Hospital Colorado

FDA approves a drug that dramatically reduces seizure frequency

Most physicians who conduct clinical trials say they were drawn to research because they wanted to discover new treatments and cures. Such findings are rare, but, every once in a while, clinical trials result in a new treatment that is life changing. This was the case with two trials led by pediatric neurologist Kelly Knupp, MD, who tested the long-term safety and tolerability of an anti-seizure medication called fenfluramine. After conducting the studies for four years, the FDA approved the drug in the summer of 2020 for the treatment of seizures associated with Dravet syndrome.

CTSA Program In Action Goals
Goal 4: Innovate Processes to Increase the Quality and Efficiency of Translational Research, Particularly of Multisite Trials