dCas9-based Scn1a gene activation restores inhibitory interneuron excitability and attenuates seizures in Dravet syndrome mice

By |2019-10-10T09:51:34-05:00October 10th, 2019|Categories: DSF Research Review|Tags: , , |

Our chromosomes are paired, and therefore we have two copies of almost every gene. The mutations that cause Dravet Syndrome affect only one copy of the Scn1a gene, which leads to production of only half the normal amount of Nav1.1 protein. New gene therapy approaches are starting to have success replacing mutated gene copies [...]

Fenfluramine HCl (Fintepla®) Provides Long-Term Clinically Meaningful Reduction in Seizure Frequency: Results of an Open-Label Extension Study

By |2019-10-10T09:44:24-05:00October 10th, 2019|Categories: DSF Research Review|Tags: , , |

This is a report on results from an open-label extension study of fenfluramine in children and young adults (2–18 years old) with Dravet syndrome. "Open-label extension" means that the participants have participated in the Phase 3 clinical trial and are continuing with the drug, but can know information that was previously blinded, such as [...]

2019 Research Grant Award Cycle

By |2020-04-06T15:31:14-05:00October 4th, 2019|Categories: Decoding Dravet Blog|

This is an exciting time of the year for DSF and our community - research grant applications were due a few weeks ago and our Scientific Advisory Board has begun reviewing this year’s applications. Announcements of our 2019 grant awardees will be made in early December, so stay tuned!  The primary reason DSF came into [...]