Providing education, support, advocacy, and promoting research
for CTX patients, their families, and medical professionals
who treat and study this rare disease.
2025 CTX Scientific & Family Meeting Agenda
12:30 – 1:25 pm | Group Lunch at Designated Tables | Burhams Dining Room |
1:30 – 1:35 pm | Welcome | Ms. Jean Pickford | Dr. Andrea DeBarber |
1:35 – 1:50 pm | Introduction to Genetic Cause and Clinical Signs & Symptoms | Dr. Bart Duell |
1:50 – 2:05 pm | Understanding CTX through Biochemical Pathways | Dr. Andrea DeBarber |
2:05 – 2:20 pm | Unveiling the Results of the Phase III RESTORE Clinical Trial | Dr. Bart Duell |
2:20 – 2:35 pm | Exploring the Diverse Clinical Spectrumof CTX | Dr. Paulo Nobrega (remote) |
2:35 – 2:50 pm | Breaking Barriers: Advancing Global Access to Treatment | Dr. Jonas Alex Saute (remote) |
2:50 – 3:05 pm | Q & A | Speaker Panel |
3:05- 3:20 pm | Break | |
---|---|---|
3:20 – 3:35 pm | Exploring Secondary Findings and the CTX Alliance Nomination Process | Dr. Robert Steiner |
3:35 – 3:55 pm | Building a CTX Patient Registry: A Path to Valuable Research Data | Dr. Robert Steiner |
3:55 – 4:15 pm | A CTX Patient Registry: A Patient’s Viewpoint | Ms. Abbey Cook |
4:15 – 4:35 pm | Pediatric Cataracts and ClinGen: Bridging Genetics and Clinical Care | Dr. Jennifer Rossen |
4:35 – 4:55 pm | CTX Alliance Update & Celebration: FDA Approval of Ctexli | Ms. Jean Pickford |
4:55 – 5:00 pm | Group Photo & Adjourn | All |