Strides in STXBP1: What Happened Last Month in STXBP1 Research?
Gene Therapy Approaches for STXBP1-Related Disorders
An Interview with Dr. Connie Smith-Hicks, Director of the Center for Synaptic Disorders at the Kennedy Krieger Institute
Shelby Miller Helped Raise Awareness for STXBP1-Related Disorders during MLB’s Players Weekend
Impressions of the 2024 STXBP1 Researcher Roundtable Meeting
STXBP1: Fast Forward to a Brighter Future
How Are We Going to Treat STXBP1 Disorders?
A Message to Our Dads from Our Own Dr John Oldenhof
An Overview of Clinical Trials
Strides in STXBP1: What Happened Last Month in STXBP1 Research?
American Society for Cell and Gene Therapy (ASCGT) conference recap by Mike Boland with Charlene Son Rigby
Preclinical Studies in Drug Development
An Introduction to Gene Therapy
Rare But Not Alone
See the Rare Diseases Day 2024 Run in Germany
Rare Diseases Day Run in Oberthal, Germany had nearly 50 runners hit the finish line
Scientific Director’s Corner: What is a Natural History Study?
We Were in the House … The White House
Hear Charlene Son Rigby’s comments on a panel discussing “Policy Challenges and Opportunities in the Rare Disease Community”
Scientific Director's Corner: Genes are the Blueprints for Making Proteins
Celebrating 2023 & Looking Forward
