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Treatment for one year or longer with the antiseizure therapy Fintepla (fenfluramine) was shown to have a minimal impact on height and weight in children with Dravet syndrome, according to data from an open-label extension study. Despite Fintepla’s active ingredient being fenfluramine — a medicine initially…

Note: This story was updated Feb. 7, 2022, to clarify that CAMP4 will continue preclinical studies for its lead candidate. Also, CAMP4 licensed the antisense oligonucleotides from OPKO Health, not the RNA Actuating Platform, which was developed in-house. CAMP4 Therapeutics‘ investigational RNA-based therapy — called CMP-SCN (CO-3527) — increased…

Zogenix is seeking approval to market Fintepla (fenfluramine) in Japan as a treatment for epileptic seizures associated with Dravet syndrome. The company’s new drug application will be reviewed and decided on by the Japanese Ministry of Health, Labour, and Welfare. “There remains a substantial unmet need in…

Vaccines for COVID-19 are generally safe for children with Dravet syndrome, and are unlikely to cause an increase in the frequency of seizures, whereas COVID-19-like disease often increases seizure frequency, according to a pair of surveys done in the U.K. Since the start of the pandemic, researchers have…

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

Single and multiple doses of Stoke Therapeutics’ investigational therapy STK-001 were well-tolerated and led to a significant reduction in the frequency of convulsive seizures among children and adolescents with Dravet syndrome in the first three months of follow-up, interim data from the ongoing Phase 1/2a MONARCH trial show.

ReS3-T, an investigational medicine developed by reMynd for therapy-resistant epilepsies such as Dravet syndrome, has been accepted into the National Institute of Neurological Disorders and Stroke (NINDS) Epilepsy Therapy Screening Program (ETSP). The ETSP identifies potential therapies for drug-resistant epilepsy and disease prevention and modification by allowing researchers…

Virpax Pharmaceuticals has acquired exclusive worldwide rights from Nanomerics to develop a nasal spray of cannabidiol to treat epilepsy in adults and children, including those with Dravet syndrome. Under the agreement, Virpax can develop, manufacture, and sell VRP324, an investigational formulation of cannabidiol (CBD) administered using a nasal…

Stoke Therapeutics’ investigational therapy STK-001 is generally safe and results in a trend toward a reduction in seizure frequency in children and adolescents with Dravet syndrome, according to interim data from the ongoing Phase 1/2a MONARCH trial. These promising early findings, concerning STK-001’s single doses up to 30…

Treatment with STK-001, Stoke Therapeutics‘ investigational therapy for Dravet syndrome, has so far raised no safety concerns among children taking part in the ongoing Phase 1/2a MONARCH clinical trial, according to the results of a planned interim analysis. Early data also hint that the medication may reduce seizure…