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