First Patient Registered for Dravet Syndrome Observational Study

According to a story from Biotech 365, the biotechnology company Stoke Therapeutics, Inc., recently announced that the very first patient has enrolled for the company’s observational study of Dravet syndrome patients. The company plans to use data from this study to aid in the development of its investigational product candidate STK-001, which is intended to treat Dravet syndrome. Stoke is focused on the development of treatments for genetic illnesses by upregulating protein expression.

About Dravet Syndrome

Dravet syndrome is a form of severe epilepsy that usually begins by the sixth month of life. The disease is most characterized by frequent seizures that are sometimes triggered by fever or hot temperatures. In most cases, the syndrome is caused by mutations of the SCN1A gene. Most of the time, these mutations are not heritable and appear spontaneously. The predominant symptom of Dravet syndrome is seizures; varying types of seizures often occur as the disorder progresses, as well as ataxia, developmental delays, behavioral disorders that may resemble autism, and cognitive impairment. Seizures can be potentially lethal. Management strategies for Dravet syndrome may include a ketogenic diet, anticonvulsant medications, cognitive rehabilitation therapy, and cannabidiol. This rare disorder is estimated to appear anywhere from 1 in every 20,000 to 1 in every 40,000 births. To learn more about Dravet syndrome, click here.

About The Study

The observational study will include patients between the ages of 2 and 18 years old. This study will last for two years and is intended to focus on both the burden of seizures (by monitoring their frequency) and measuring the impact of other symptoms as well, such as sleep problems, intellectual disability, and more. The study will provide enrollment at 20 different sites across the US. The patients are not required to halt their treatment during the study and will continue to receive their regular care.

Although this study will not involve the use of STK-001 or any other experimental treatment, the data from it can provide critical information related to the course of the disease that can inform the development of new therapies and allow researchers to quantify the impact of symptoms in the daily lives of patients.


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