STXBP1 Summit+ 2021 Conference Recordings

Captions are available to all our Scientific session video recordings to assure that they are accessible to everyone!

Scientific Stage Sessions

Science

Session #1
STXBP1 Welcome
Clinical Update

Session #2
Genetics 101
Designing an Approach to Measuring Development in STXBP1
Natural History of STXBP1 in Adulthood

Translational Research

Session #3
Mouse and Organoid Models for Studying STXBP1 Disorders
New Insights in Disease Mechanisms and New Drug Screening

Therapy Development

Session #4
Clinical Trial Readiness
Gene Therapy

Session #5
Antisense Oligonucleotides (ASOs)
Small Molecule Therapies
4-Phenylbutyrate Clinical Trial Update

Natural History Studies

Session #6
Simons Searchlight & STXBP1 Study Update
Ciitizen Natural History Study