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New Gene Therapy May Potentially Treat Severe Form of Epilepsy

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.
Ericr Wengert Manojk Patel School Of Medicine University Of Virginia Augmentation Of Nuclear Gene Output Dravet Syndrome In Brain Research

Newly developed gene therapy can treat a severe form of epilepsy and prolong survival

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.
Ericr Wengert Manojk Patel Emily Henderson Augmentation Of Nuclear Gene Output Drug Administration University Of Virginia Health System

Stoke Therapeutics Presents Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2021 Annual Meeting

– 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
United Kingdom United States Elaine Wirrell Helen Cross Barry Ticho Colin Roberts

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Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates

Press release content from Business Wire. The AP news staff was not involved in its creation. Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates May 10, 2021 GMT BEDFORD, Mass.--(BUSINESS WIRE)--May 10, 2021-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today reported financial results for the first quarter of 20...
United States Borough Of Bedford United Kingdom Edwardm Kaye Eric Rojas Augmentation Of Nuclear Gene Output
Source: apnews.com

Stoke Therapeutics Reports Full Year 2020 Financial Results and Provides Business Updates

Press release content from Business Wire. The AP news staff was not involved in its creation. Stoke Therapeutics Reports Full Year 2020 Financial Results and Provides Business Updates March 9, 2021 GMT BEDFORD, Mass.--(BUSINESS WIRE)--Mar 9, 2021-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today reported financial results for the full year ended De...
United States United Kingdom Edwardm Kaye American Epilepsy Society Augmentation Of Nuclear Gene Output Stoke Therapeutics Inc
Source: apnews.com

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