May 17-19, 2022
Delivered as a Hybrid EventBoston, MADAY 2 ✦ IN-PERSON ✦ Tue, Dec 7keyboard_arrow_right
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DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 - EDT
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DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 - EDT
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DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 8:30am - 9:00am30 mins
Continental Breakfast and Networking
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 9:00am - 9:15am15 mins
Chairman’s Welcome and Day 1 Review
- Tom Croce - Vice President, Patient Advocacy, bluebird bio
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 9:15am - 10:00am45 mins
TECHNOLOGY SPOTLIGHT ● Developing and Scaling Digital Therapeutics for Rare Diseases
- Everett Crosland - Chief Commercial Officer, Cognito Therapeutics
- Melinda Decker - Chief Commercial Officer, Mymee
- Mark Grossman - Vice President, Business Development, Gomo Health
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 10:00am - 10:45am45 mins
CUTTING-EDGE CASE STUDY ● Leverage Genetic Testing for Value for Rare Disease Patients
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 10:45am - 11:15am30 mins
Networking and Refreshment break
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 11:15am - 12:00pm45 mins
Reflect on the Impact of COVID-19 on the Rare Disease Community
- Scott Williams - Head, Global Public Affairs, Rare Diseases and Rare Blood Disorders, Sanofi Genzyme
- Jocelyn Duff - Founder & CEO, CureCMT4J
- Sanjeev Luther - President and CEO, Rafael Pharmaceuticals
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 12:00pm - 12:45pm45 mins
Identify Gaps and Develop Solutions to Promote Diversity, Equity and Inclusion
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 12:45pm - 1:45pm60 mins
Networking Luncheon
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 1:45pm - 2:30pm45 mins
Fostering Effective Relationships Between Industry and Advocacy
- Jennifer Helfer - Senior Director, Patient Advocacy & Engagement, Encoded Therapeutics Inc.
- Chelsey Hathaway McCarthy - Executive Director, The DDX3X Foundation
- Monica Fay - Senior Vice President, Medical Affairs, Apellis Pharmaceuticals
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 2:30pm - 3:15pm45 mins
Harness the Power of Data to Drive Progress
- Alison Skrinar - Vice President, Clinical Outcomes Research & Evaluation, Ultragenyx Pharmaceuticals
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 3:15pm - 3:45pm30 mins
Networking and Refreshment break
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 3:45pm - 4:30pm45 mins
Optimize Decentralized Trials to Support the Future of Rare Disease Research
- Jim Palma - Executive Director, TargetCancer Foundation
DAY 2 ✦ IN-PERSON ✦ Tue, Dec 7 4:30pm - 5:00pm30 mins
Risks and Rewards of Rare Disease Drug Development — Highlight Opportunities and the Way Forward
- John Maslowski - Chief Commercial Officer, Forge Biologics
- Martine Zimmermann - Global Head of Regulatory Affairs, Alexion Pharmaceuticals