10/29/2025
Sending a heartfelt thank you to National Organization for Rare Disorders, Inc. (NORD) for hosting us at last week’s inspiring Rare Diseases and Orphan Products Breakthrough Summit! 🦓💙 We were grateful for the opportunity to join meaningful discussions on tough topics surrounding CGT accessibility, acceptability, and affordability—and to celebrate the success stories that remind us what’s possible when innovation meets hope.