Designation based on preliminary clinical evidence indicating the potential for ETX101 to address unmet medical needs associated with SCN1A+ Dravet syndrome Completion of enrollment in POLARIS ...
The SCN1A gene is too large to fit into aconventional viral delivery systems. Scientists overcame this hurdle with a pioneering "split-intein" mechanism, which is like two separate trucks, delivering ...
7monon MSN
Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
Dravet syndrome affects around 1 in 15,700 children, and most cases are caused by mutations in the SCN1A gene. This gene ...
Scientists from the University of Virginia (UVA) School of Medicine describe in a study involving mouse models how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics today announced the upcoming presentation of initial data from the ENVISION prospective natural history study of children with SCN1A+ ...
Scientists from the Allen Institute and Seattle Children’s Research Institute have announced a breakthrough in the development of gene replacement therapies for Dravet syndrome, a rare form of ...
SEATTLE, Wash. -- Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is a rare and debilitating form of epilepsy that starts ...
A lifelong illness, Dravet syndrome develops because of a genetic mutation that affects the ability of brain cells to send and receive messages. About 1 in 15,700 infants are born with Dravet syndrome ...
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