Select Publications and Presentations

Optimized human regulatory sequences achieve targeted expression in brain and decreased off-target liver and dorsal root ganglion expression

Optimized Human Regulatory Sequences Achieve Targeted Expression in CNS and Decreased Liver Expression in Mice

Efficient Delivery of Clinically-Validated Adeno-Associated Viral (AAV) Vectors to the Central Nervous System: A Systematic Evaluation of Multiple Routes of Administration and Viral Serotypes in Cynomolgus Macaques

Behavior, social-emotional, and sleep difficulties in young children with SCN1A+ Dravet Syndrome Participating in the ENVISION study, an international, prospective natural history study

The ENVISION Study, an international, prospective natural history study in young children with SCN1A+ Dravet syndrome, 18-month follow-up

The ENVISION Study, an international, prospective natural history study in young children with SCN1A+ Dravet syndrome

The ENVISION Study, an International, Prospective Natural History Study in Young Children with SCN1A+ Dravet Syndrome

ENVISION: An International, Prospective Natural History Study in Young Children with SCN1A+ Dravet Syndrome

Molecular Diagnostic and Phenotypic Findings in Individuals With SCN1A-Related Disease Identified Through a Sponsored, No-Charge Epilepsy Genetic Testing Program

A Patient-Oriented Approach to Integrating Families’ Experiences in Clinical Trials Design and Clinical Care for Dravet Syndrome

DRAVET ENGAGE. Parent Caregivers of Children with Dravet Syndrome: Perspectives, Needs, and Opportunities for Clinical Research

The Clinical, Economic, and Humanistic Burden of Dravet Syndrome — A Systematic Literature Review

ETX101, a GABAergic Interneuron Selective AAV-mediated Gene Therapy for Treatment of SCN1A+ Dravet Syndrome: Biodistribution and Safety in Non-human Primates

Reducing the time to diagnosis and increasing the detection of individuals with SCN1A-related disease through a sponsored epilepsy genetic testing program

From Gene Replacement to Gene Regulation: Developing a Disease-Modifying AAV Gene Therapy Vector for SCN1A-Positive (SCN1A+) Dravet Syndrome

Proposed Design for a Natural History Study of SCN1A-Positive (SCN1A+) Dravet Syndrome

A GABA-Selective AAV Vector-Based Approach to Up-Regulate Endogenous Scn1a Expression Reverses Key Phenotypes in a Mouse Model of Dravet Syndrome