Veronica Hood, PhD

DSF recently announced funding for a Longitudinal Dravet Syndrome Natural History Study led by Dr. Kelly Knupp with The Dravet Lifespan Multidisciplinary Clinic at Children’s Hospital Colorado. Today’s blog explores the importance of understanding the natural history of a disease, ways natural history data are collected, and an overview of some of the past and […]

Scientific advances in gene and cell therapy are moving fast. CRISPR-based therapies are being developed in record time to help children with life-threatening conditions. At the same time, new delivery approaches are making it easier to target the brain for neurological diseases. As the FDA and other regulators shift leadership and strategy, the entire landscape

A recent medical breakthrough resulted in the successful treatment of a child with a rare genetic disorder with a personalized CRISPR gene editing therapy developed at lightning speed thanks to the work of researchers at Children’s Hospital of Philadelphia (CHOP). It’s the kind of story that brings hope to rare disease communities watching from the

A recent publication in the journal of Science Translational Medicine revealed a groundbreaking new approach to a genetic therapy for Dravet syndrome. The new scientific approach was developed and validated through a collaboration between researchers at Allen Institute for Brain Science and University of Washington’s Seattle Children’s Research Institute. The gene therapy was tested in

For nearly 25 years now we have known that mutations in the sodium channel gene, SCN1A, can lead Dravet syndrome and related epilepsies. Since the initial discovery, over 1,700 unique mutations occurring across the length of the large SCN1A gene have been identified in patients with Dravet syndrome. The awareness and availability of genetic testing

Currently, the ARGUS Trial is enrolling participants with Dravet syndrome to evaluate if Clemizole (EPX-100) can reduce the number of seizures. Clemizole has a long history, from its use as an antihistamine to the exciting discovery (in zebrafish!) that it may be effective at reducing the number of seizures in Dravet syndrome. Today’s blog delves

This is an exciting time for the Dravet syndrome community. The first potentially disease-modifying treatment targeting the genetic cause of Dravet syndrome has completed initial human studies and is now advancing to a pivotal global Phase 3 trial. If the study confirms that this therapy is safe and effective, the next step will be seeking

Dravet Syndrome Foundation (DSF) was established in 2009 with a mission largely focused on funding and advocating for Dravet-related research. To date, DSF has directed over $10.5 million to support research. DSF’s Research Grant Programs provide essential funding for research focusing on Dravet syndrome and related disorders. These grants support early-stage research endeavors exploring hypotheses

Long-term outcomes for patients with Dravet syndrome can vary quite a bit. Some patients may have more significant impacts on cognition or speech, while others may struggle most with behavioral or sleep challenges. There can also be differences in the age of onset, the types of seizures that are present, the severity of seizures, and

Earlier this December, DSF Staff and Board Members attended the 2024 American Epilepsy Society Meeting to learn about the exciting advancements occurring in epilepsy research, make connections and cement current collaborations, advocate for and seek out new research opportunities, and raise awareness of Dravet syndrome and the work of DSF and the patient-community to support