Agenda
12:00 PM – 1:00 PM EDT | Welcome & Networking Lunch
1:00 PM – 4:30 PM EDT | Rare Disease Symposium Program
- The Patient View
- David Downing, GRIN1 Dad
- Jaime McHugh, Rare Disease Mom and NORD Running for Rare Champion
- The Research View
- Dr. Shira Rockowitz, PhD, Data Science Director, Boston Children’s Hospital, Children's Rare Disease Collaborative Co-Leader
- Dr. Piotr Sliz, PhD, Vice President, Chief Research Information Officer & Associate Professor, Boston Children’s Hospital, Children's Rare Disease Collaborative Co-Leader
- The FDA View
- Amy Rick, Director of Strategic Coalitions for FDA’s Rare Disease Innovation Hub
- The Policy View
- Karin Hoelzer, Senior Director, Patient Advocacy, BIO
- Jack Kalavritinos, Founder, JK Strategies and the Washington Health Innovation Council, and Former Director, HHS Office of Intergovernmental & External Affairs
- Judy Stecker, SVP, Burson, and Former HHS Deputy Chief of Staff for Strategy & Operations – Rare Disease Parent & Founder, Wheeler’s Warriors
- The View from the National Organization for Rare Disorders
- Pamela Gavin, Chief Executive Officer, NORD
- The View from the Rare As One Network
- Heidi Bjornson-Pennell, Senior Program Manager, Science in Society, and Lead, Rare As One Network
- The Biotech CEO View
- Paula Ragan, PhD, CEO, X4 Pharmaceuticals
4:30 PM – 5:30 PM EDT | Networking Reception
