World Orphan Drug Congress

Join us in Boston for three days of the most comprehensive program and inclusive gathering of rare disease stakeholders.

Round table discussion:

Wednesday, April 23^rd 12:05 – 12:50 pm, Theater 7

“Gene Therapy for Rare Diseases – Clinical Trial Experiences and Lessons Learned”

Speakers:

• Mariana Burin, Medical Director
• Elizabeth Donahue, Sr. Director, Project Management

Join us for a Round Table where we’ll discuss:

• Challenges in designing gene therapy clinical trials
• Lessoned learned around long-term follow up and the need for ongoing monitoring
• Incorporating feedback from patients and families into trial design
• Key considerations for gaining approval from regulatory bodies