DRAVET SYNDROME (DS)
Clinical trials for DRAVET SYNDROME (DS) explained in plain language.
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Gene therapy hopes to tame severe childhood epilepsy
Disease control Recruiting nowThis early-stage trial tests a gene therapy called RC001 in children aged 2 to 18 with Dravet syndrome, a severe form of epilepsy caused by a genetic mutation. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and how the body proc…
Matched conditions: DRAVET SYNDROME (DS)
Phase: EARLY_PHASE1 • Sponsor: Second Affiliated Hospital of Guangzhou Medical University • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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New hope for babies with severe epilepsy: fenfluramine safety trial launches
Disease control Recruiting nowThis study is testing the safety of fenfluramine, a drug already approved for children 2 and older with Dravet syndrome, in younger children aged 12 to 23 months. Dravet syndrome is a severe genetic epilepsy that starts in the first year of life. The study will enroll 5 children …
Matched conditions: DRAVET SYNDROME (DS)
Phase: PHASE4 • Sponsor: University of Colorado, Denver • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC