The Scientific Director’s Corner

Hello, here you can read various blog posts from me and Foundation interns on subjects that we hope our families find interesting and informative.

And, you can also ask me questions or suggest topics you would like covered in the future using the Ask the Scientific Director form below.
James R. Goss, PhD


Strides in STXBP1

Monthly update on new papers related to STXBP1.

Aug/Sep 2025
June/July 2025
May 2025
April 2025
March 2025
January / February 2025



Science Saturdays Videos

Introducing Jim

What is a Natural History Study?

An Introduction to Gene Therapy

The Role of Preclinical Studies in Drug Development

Coming soon
An Overview of Clinical Trials

How are we going to treat STXBP1 disorders

Using patient medical electronic medical records to conduct natural history studies

Gene Therapy Approaches to Treat STXBP1-RD


Ask the Scientific Director