A molecule designed to enhance activity at certain GABA-A receptor proteins in the brain protected against seizures and eased anxiety in a mouse model of Dravet syndrome, recent research showed. At higher doses, the medication led to sedative side effects, which scientists believe can be avoided by making sure…
News
Behavioral development in children and teens with Dravet syndrome generally held stable in the first year of an observational study, though some showed small improvements in communication, according to researchers. Participants in the study, dubbed BUTTERFLY, continued to perform below what would be expected of their typically developing peers…
Encoded Therapeutics has been cleared by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin clinical testing of ETX101, an investigational one-time gene therapy for children with Dravet syndrome. A Phase 1/2 clinical study dubbed EXPEDITION will test how safe ETX101 is and how well it…
The National Institute of Drug and Food Surveillance (INVIMA) of Colombia has approved Trunerox as an add-on treatment for seizures in people with Dravet syndrome and Lennox-Gastaut syndrome (LGS). Trunerox, which was developed by the Canadian biopharmaceutical Avicanna, is a proprietary oral formulation of 10% cannabidiol…
Encoded Therapeutics has received clearance from U.S. and Australian regulatory agencies to launch Phase 1/2 clinical trials testing ETX101, its Dravet syndrome gene therapy candidate, in infants and young children with the seizure disorder. In the U.S., the company plans to begin ENDEAVOR (NCT05419492), which will be…
International Epilepsy Day for 2024 aims to promote public understanding of a condition thought to affect some 65 million people globally — including those with Dravet syndrome — to gather support for a 10-year World Health Organization (WHO) epilepsy roadmap. Held annually on the second Monday in February —…
Fintepla (fenfluramine) eased brain inflammation, protected nerve cells from damage and death, and extended survival in a mouse model of Dravet syndrome, a study showed. “These results support clinical observations that [Fintepla] may have benefits beyond seizures,” its scientists wrote. The article, “Fenfluramine increases survival…
Real-world use of Fintepla (fenfluramine) was found to be safe and effective for people with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), according to a recent report from Spain. Data, which showed that 60.3% of Dravet patients experienced a clinically meaningful reduction in seizures, are generally consistent with…
Treatment with Fintepla (fenfluramine) appeared to be reasonably safe and to effectively lower seizure frequency in five young children with Dravet syndrome who started on the therapy before they were 2 years old, scientists in Italy reported. These findings are especially noteworthy because, in…
Most Dravet syndrome patients utilize various types of therapies beyond medicines to manage the disease, including physiotherapy, occupational therapy, and psychological therapy, according to a recent survey of caregivers and family members in Spain. While survey respondents sometimes found these non-pharmacological interventions to be effective, sizable numbers were less…
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