The Dravet Syndrome Foundation (DSF) is a non-profit 501(c)(3) organization founded in 2009 dedicated to aggressively funding research for treatments and a cure for Dravet syndrome and other sodium channel encephalopathies. The foundation serves as the primary patient advocacy organization for the Dravet community in the United States, bridging patients, families, researchers, and industry to accelerate therapeutic development.
To fund research for treatments and a cure for Dravet syndrome and related epilepsies, while providing support and education to patients, families, and caregivers affected by these conditions.
DSF is one of the primary non-governmental funders of Dravet syndrome research, providing:
- Research grants: Annual grant cycles funding basic science, translational, and clinical research
- Natural history study support: Funding and patient recruitment for natural history registries that are critical for clinical trial design
- Preclinical consortium: Collaborative programs that share data and resources across academic labs working on Dravet
- Biomarker development: Support for development and validation of biomarkers needed for clinical trial endpoints
The foundation plays a critical role in connecting patients with clinical trials:
- Trial matching: Direct patient-to-trial referrals through the foundation's patient registry
- Site identification: Working with companies to identify suitable clinical trial sites
- Patient education: Informing families about trial eligibility, risks, and expectations
- Advocacy for access: Supporting policies that facilitate patient access to investigational therapies
¶ Patient and Family Support
- Family conference: Annual family conference bringing together patients, families, researchers, and industry
- Family support programs: Emergency funds, equipment assistance, and respite care programs
- Education: Webinars, educational materials, and guidelines for managing Dravet syndrome
- Registry: Patient registry with genetic data and clinical information enabling research recruitment
DSF engages with regulatory agencies to advocate for patient-centered drug development:
- FDA engagement: Participating in FDA Patient-Focused Drug Development meetings
- Orphan drug designation: Supporting companies seeking orphan drug designation and rare pediatric disease PRVs
- Accelerated approval: Advocating for pathways that enable faster access to transformative therapies
- International harmonization: Working with international regulatory counterparts on harmonized approaches
DSF maintains collaborative relationships with multiple companies developing Dravet therapies:
DSF supports the primary natural history studies used as external controls in clinical trials:
Dravet Syndrome Natural History (RDCRN DM1B):
- Multi-center observational study
- N=500+ patients enrolled
- Characterizes seizure trajectory, developmental outcomes, and mortality
- Endpoints validated for use as natural history comparators
Genesis Dravet (Taysha/UCB):
- Industry-sponsored natural history study
- N=200 patients
- Focus on developmental plateau and SUDEP risk characterization
The foundation's activities have directly impacted the Dravet therapeutic landscape:
- Enabled clinical trials: Patient registry and site support enabled rapid enrollment in STK-001 and ETX101 trials
- Natural history as external control: Foundation-funded studies provide the comparator data for single-arm accelerated approval submissions
- Biomarker validation: Foundation support for EEG and genetic biomarker studies informed endpoint selection
- Regulatory engagement: Foundation testimony contributed to FDA's understanding of caregiver burden and unmet need
- International Dravet Syndrome Epilepsy Foundation (IECF) — Global umbrella organization
- Epilepsy Foundation — Broader epilepsy advocacy
- Rare Epilepsy Network (REN) — RDCRN consortium
- Loulou Foundation — CDKL5 deficiency advocacy
- Angelman Syndrome Foundation — Angelman syndrome advocacy
- Cure STXBP1 — STXBP1 encephalopathy advocacy
- PCDH19 Alliance — PCDH19 advocacy