An experimental disease-modifying drug being developed by Stoke Therapeutics and Biogen helped reduce seizures and improve quality of life for children and teenagers with Dravet syndrome, a rare but devastating genetic form of epilepsy, in early and mid-stage trials.
Children who regularly took zorevunersen for up to three years experienced up to 91% fewer seizures, with monthly episode counts falling from 17 to between 1.5 and 7, depending on the drug regimen tested, researchers reported in The New England Journal of Medicine.
Eighty-one patients ages 2 to 18 received between 10 milligrams and 70 mg of zorevunersen injected directly into the cerebrospinal fluid, either as a single dose or with additional doses two or three months later, over the course of six months.
Seventy-five of the 81 took part in second-stage trials and continued to receive the drug every four months.
Zorevunersen was generally well tolerated, and most side effects were mild to moderate, researchers found.
Dravet syndrome is notoriously hard to treat and affects about 1 in 15,000 children.
With Dravet, one copy of the patient's two SCN1A genes fails to produce enough protein for nerve cells to function properly, resulting in developmental delays, coordination and eating problems, severe seizures, and early death.
Zorevunersen works by increasing the protein production of the patient's healthy copy of the gene.
Larger trials of the drug are underway.
"I regularly see patients with hard-to-treat genetic epilepsies with impacts that go beyond seizures and it's heartbreaking when treatment options are limited," study leader Helen Cross of University College London said in a statement.
The mother of an 8-year-old named Freddie who participated in the trial said in a statement that it "has completely changed our lives."
"We now have a life we didn't ever think was possible and most importantly it's a life that Freddie can enjoy."
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