Gene Therapy Approaches for STXBP1-Related Disorders

Written By Russ Novy

Watch this short video on our YouTube channel

Gene therapy holds significant promise for improving the lives of individuals with STXBP1-related disorders. Currently, researchers and biopharma companies are developing several gene therapy approaches for treating STXBP1-RD.

Our Scientific Director, James Goss, has created a presentation to help parents understand gene therapy, summarize the various gene therapy approaches being developed for STXBP1-RD - including their pros and cons - and discuss the challenges of conducting gene therapy clinical trials for this rare disorder.

This short video covers:

  • Background on genes and proteins

  • Gene therapy approaches under development right now

  • Challenges for gene therapy drug approvals in rare diseases like STXBP1-related

Russ Novy
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