DRAVET SYNDROME
Clinical trials for DRAVET SYNDROME explained in plain language.
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Gene therapy trial aims to tame severe seizures in kids
Disease control OngoingThis study tests a gene therapy called ETX101 in 4 children with Dravet syndrome, a severe epilepsy disorder. The treatment uses a harmless virus to deliver a working copy of the SCN1A gene. The goal is to see if it safely reduces seizures and improves communication skills.
Matched conditions: DRAVET SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated May 26, 2026 09:46 UTC
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New hope for babies with severe epilepsy: drug trial targets dravet syndrome
Disease control OngoingThis study tests the safety of fenfluramine in 25 infants aged 1 to 2 years with Dravet syndrome, a severe form of epilepsy. The medicine is given daily alongside other seizure drugs to help control seizures. The goal is to see if it is safe and well-tolerated in this young age g…
Matched conditions: DRAVET SYNDROME
Phase: PHASE3 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated May 26, 2026 09:34 UTC
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New hope for dravet syndrome: expanded access to seizure drug
Disease control APPROVED_FOR_MARKETINGThis program provides expanded access to stiripentol for people with Dravet syndrome, a severe form of epilepsy that causes frequent, hard-to-control seizures. It is open to patients aged 6 months and older who have not responded to other treatments. The goal is to help reduce se…
Matched conditions: DRAVET SYNDROME
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Disease control
Last updated May 26, 2026 09:33 UTC
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Gene therapy breakthrough for rare childhood epilepsy?
Disease control OngoingThis study tests a new gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The treatment aims to reduce seizures and improve development by delivering a working gene to brain cells. The study is early-stage (Phase 1/2) and focuses on safety, …
Matched conditions: DRAVET SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated May 26, 2026 09:32 UTC
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Hope for dravet syndrome: experimental drug moves to Long-Term safety check
Disease control OngoingThis study looks at the long-term safety of an experimental drug called zorevunersen (STK-001) in 60 people with Dravet syndrome who have already taken the drug in earlier studies. Researchers will monitor side effects, seizure frequency, and overall well-being. The goal is to se…
Matched conditions: DRAVET SYNDROME
Phase: PHASE2 • Sponsor: Stoke Therapeutics, Inc • Aim: Disease control
Last updated May 26, 2026 09:27 UTC
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New hope for dravet syndrome: expanded access to fenfluramine offers seizure control
Disease control APPROVED_FOR_MARKETINGThis study provides fenfluramine to people with Dravet syndrome whose seizures are not controlled by other medications. The goal is to make the drug available and check its safety. Participants must have a genetic diagnosis and have tried all standard seizure drugs.
Matched conditions: DRAVET SYNDROME
Sponsor: University of California, Los Angeles • Aim: Disease control
Last updated May 20, 2026 11:58 UTC
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Epidiolex liver check: study tracks long-term injury risk
Knowledge-focused OngoingThis study checks if long-term use of Epidiolex (cannabidiol) can cause liver damage or scarring in people with certain seizure disorders. It includes 154 participants who already take or will start Epidiolex for Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis com…
Matched conditions: DRAVET SYNDROME
Phase: PHASE4 • Sponsor: Jazz Pharmaceuticals • Aim: Knowledge-focused
Last updated May 26, 2026 09:34 UTC
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New study aims to uncover hidden risks of sudden death in dravet syndrome
Knowledge-focused OngoingThis study looks at how repeated seizures may harm breathing and heart control in people with Dravet syndrome, a severe form of epilepsy. Researchers will monitor 92 children and adults over 24-48 hours to measure changes in breathing and heart rate during and between seizures. T…
Matched conditions: DRAVET SYNDROME
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated May 26, 2026 09:27 UTC