Skip to main content

Tools with a Mission: Using Disease Models to Find a Treatment for PURA Syndrome

Published Date

Jack’s Tomorrow and the JAX Rare Disease Translational Center (RDTC) are partnering to accelerate research into PURA syndrome, a rare neurodevelopmental disorder with no available treatment options. This short webinar explores the urgent need for innovative models and highlights JAX’s development of iPSC lines to advance understanding of PURA syndrome and enable drug discovery. These efforts aim to provide researchers and pharmaceutical partners with powerful tools to uncover disease mechanisms and develop new therapies.

In this webinar, you will learn:

  • What PURA syndrome is, its impact on families, and why advancing the study of rare diseases like PURA syndrome matters for future therapies
  • How the RDTC is creating iPSC models for PURA syndrome and making them available to the research community
  • How these models are being used to uncover disease biology and accelerate therapeutic development