Patient and Family Perspectives help to Unlock CTX

On September 14th, 2021 the ULF hosted a Patient Focused Drug Development (PFDD) meeting with the FDA. This meeting gave CTX patients a voice to speak directly to the FDA about their daily lives living with CTX, in the hopes that their stories will impact current drug therapies and their use, and drug development in the future. On this page, you can view the entire meeting recording and the Voice of the Patient report that shares the learnings from the meeting.

View the Recording:


View the Voice of the Patient Report

Thank you to our Partner Organizations!