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Most Dravet syndrome epilepsy cases are caused by a mutation in the SCN1A gene, resulting in a reduction in SCN1A protein production. A novel therapeutic approach, called TANGO, is designed to boost production of SCN1A to normal levels. If successful, it would be the first treatment for the fundamental cause of the disease, a lack of this particular protein in specialized brain cells.
Research from the University of Virginia School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition.
Research from the University of Virginia School of Medicine suggests how a gene therapy can treat Dravet syndrome, a severe form of epilepsy.
– Single doses of STK-001 up to 30mg and multiple doses of 20mg were well tolerated with no safety concerns related to study drug – – 70.6% of patients treated with STK-001... | December 3, 2021
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