For decades, the treatment of developmental and epileptic encephalopathies has focused on suppressing seizures rather than modifying the underlying biology of disease. In the New England Journal of ...
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced that it will present encore open-label extension data from the company’s investigation of EPX-100 (clemizole hydrochloride) in the ...
– New data include 24-month results from the largest and longest prospective natural history study of children and adolescents with Dravet syndrome, which showed a lack of improvement despite ...
The Dravet syndrome community is invited to share videos highlighting the sounds of their everyday life with fewer seizures on FINTEPLA ® (fenfluramine). The project will display a gallery of videos ...
Most parents worry about scraped knees, tumbles on the playground or gym class injuries. But for the Raia and Tisdale families, their everyday fears are life-threatening: a seizure that can strike ...
A novel first-in-class gene-regulation therapy for Dravet syndrome was associated with large reductions in seizures and improved functional outcomes in children with Dravet syndrome. Results from ...
Stoke Therapeutics' TANGO platform enhances RNA output to compensate for gene mutations causing severe genetic diseases. STOK primarily focuses on developing treatments for Dravet syndrome and ...
Add Yahoo as a preferred source to see more of our stories on Google. When you buy through links on our articles, Future and its syndication partners may earn a commission. 12-year-old Preston Raynor ...
Takeda Drug Fails Pivotal Tests in 2 Rare Epilepsies; Path Forward Depends on Regulatory Flexibility
A Takeda Pharmaceutical drug in development for rare forms of epilepsy that are resistant to anti-seizure medications has failed to meet the main goals of two placebo-controlled pivotal studies, a ...
Fenfluramine, a drug that was previously sold as an appetite suppressant before it was withdrawn from the market in 1997 over concerns about its link to heart valve problems, may have a new ...
After raising $52,000 with a 5K last year to fund research into their son’s rare seizure disorder, Matthew and Reagan Hill hope for similar success at “Cael’s Climb” at McDade Park on Sunday. Cael ...
Encoded Therapeutics, Inc. ("Encoded"), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the U.S. Food and Drug ...
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