Research News
Voice of the Patient report Missed our Externally-led Patient Focused Drug Development (EL-PFDD) Meeting on Dravet Syndrome on February 3, 2022? We are now working on our Voice of the Patient report, which will be developed based on the content of the meeting and sent to the FDA and housed on the DSF website. This …
Particpate in the EL-PFDD on Dravet Syndrome Externally-Led Patient Focused Drug Development (EL-PFDD) Meeting on Dravet Syndrome EL-PFDD meetings bring together patients and caregivers, US Food and Drug Administration (FDA) representatives, pharmaceutical companies, and doctors who are experts in the particular disease. DSF will be hosting a virtual EL-PFDD on Dravet syndrome on February 3, …
2021 DSF Grant Awardees DSF is dedicated to funding the highest caliber research on Dravet syndrome and associated epilepsies. Our focus is on research projects that will find new treatments and improve the quality of life for those living with an ion channel epilepsy. We place a high priority on funding research that has a …
AES 2021 Meeting Summary On December 3-7th, 2021, the American Epilepsy Society (AES) held its annual conference to bring together clinicians and scientists on the cutting edge of epilepsy research. This year had a multitude of presentations related to Dravet syndrome, making for an exciting few days focused on what we have learned about Dravet …
Wenxi Yu, PhD – University of Michigan $50,000 ONE YEAR POSTDOCTORAL FELLOWSHIP Co-funded with JAM for Dravet Mutations in the sodium channel genes SCN1A and SCN8A are a significant cause of Developmental Epileptic Encephalopathies (DEEs), severe seizure disorders. We demonstrated that reduced expression of Scn8a using a specific ASO can prevent the onset of seizures …
Optimizing the Regional Administration of SCN8a-targeting RNAi Therapy Read More »
Simona Balestrini, MD, PhD – UCL Queen Square Institute of Neurology Sanjay Sisodiya, PhD, FRCP – UCL Queen Square Institute of Neurology $150,000 TWO YEAR CLINICAL RESEARCH AWARD Dravet syndrome (DS) is caused by a genetic change that leads to severe epilepsy with difficult-to-treat seizures, cognitive impairment, other neurological and physical symptoms, and heightened risk …
Liu Lin Thio, MD, PhD – Washington University in St. Louis Joel R. Garbow, PhD – Washington University in St. Louis $150,000 TWO YEAR RESEARCH AWARD Co-funded with JAM for Dravet Children with Dravet syndrome have drug-resistant epilepsy characterized by different seizure types along with developmental regression and intellectual impairment. Dravet syndrome is genetic with …
Ketogenic Diet Modulated Brain Energy Metabolism in Dravet Syndrome Read More »
Jacy Wagnon, PhD – Ohio State College of Medicine $150,000 TWO YEAR RESEARCH AWARD Many individuals with Dravet syndrome (DS) do not achieve adequate seizure control using available drug treatments. These drugs also do not sufficiently treat other symptoms of DS, including behavioral and cognitive impairments. We analyzed gene expression in a mouse model of …
Targeting Molecular Responses to Seizures in Dravet Syndrome Read More »
Manisha N. Patel, PhD – University of ColoradoKelly G. Knupp, MD – Children’s Hospital Colorado $150,000 TWO YEAR RESEARCH AWARD Dravet syndrome (DS) is a catastrophic developmental and epileptic encephalopathy characterized by intractable early-life seizures, and debilitating comorbidities such as cognitive deficits, developmental delay, sleep disturbances, progressive movement abnormalities and increased risk of sudden unexpected …
Lymphoblast cell lines as a model to uncover metabolic defects in Dravet syndrome Read More »
Phase 1/2a MONARCH clinical trial of STK-001 update Stoke Therapeutics released interim safety, pharmacokinetic, and CSF exposure data from their Phase 1/2a MONARCH trial of STK-001 in individuals with Dravet syndrome. • The data in this analysis comes from 21 patients in the Single Ascending Dose arm of the study (SAD: 1 dose with a …
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