March 21-23, 2023
Delivered as a Hybrid EventPhiladelphia Marriott Downtownkeyboard_arrow_right
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DAY 1 ✦ IN-PERSON ✦ Mar 21 - EST (Eastern Standard Time, GMT-5)
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DAY 1 ✦ IN-PERSON ✦ Mar 21 - EST (Eastern Standard Time, GMT-5)
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8:00am - 8:30am30 mins
Continental Breakfast and Networking
8:30am - 8:45am15 mins
General Session
Informa Connect and Chair Welcome Remarks
- Craig Martin - Board Director, President, Global Genes, Rithim Health
- Callie Mertel - Conference Producer, Informa Connect
8:45am - 9:30am45 mins
General Session
Keynote Address from CEO
- Ethan Perlstein - CEO, Perlara PBC
9:30am - 10:15am45 mins
General Session
Patient Perspective Keynote
- David Rose - Business Development, Sales Associate, RARE Revolution Magazine
10:15am - 10:45am30 mins
Networking and Refreshment Break
10:45am - 11:30am45 mins
General Session
Navigate FDA Updates on Rare Disease Product Development
- Janet Maynard - Director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, U.S. Food and Drug Administration
11:30am - 12:15pm45 mins
General Session
Establish and Navigate the Relationship between Industry and Advocacy
- Sanjeev Luther - President and CEO, Cornerstone Pharmaceuticals
- Kari Rosbeck - President & CEO, TSC Alliance
- Amy Akers Teets - Global Public Affairs Lead, Pompe & MPS, Sanofi
- Tom Croce - Vice President, Global Patient Advocacy & Engagement, Jazz Pharmaceuticals
12:15pm - 1:30pm75 mins
Networking Luncheon
1:30pm - 2:15pm45 mins
General Session
Integrating Racial Disparity into the Rare Disease Conversation
- Mary McGowan, MHRM - CEO, Foundation for Scardiosis Research
- Lolita Smith - Program Manager, Rare Disease Diversity Coalition, Black Women's Health Imperative
- Michelle Rivas - Senior Manager, Product Communications, Horizon Therapeutics
2:15pm - 3:00pm45 mins
General Session
Understand the Patient Barriers Within Rare Disease
- Jonathan Cabral - US Head, Patient Services and Market Access, NS Pharma
- Sanjeev Luther - President and CEO, Cornerstone Pharmaceuticals
- Lisa Safarty - VP, Community Engagement, National Organization for Rare Disorders (NORD)
3:00pm - 3:30pm30 mins
Networking and Refreshment Break
3:30pm - 4:15pm45 mins
General Session
Identifying Rare Disease Patients—Finding a Needle in a Haystack
- Robert Neusner - Director, Global Commercial Strategy, Precision Medicine, The Janssen Pharmaceutical companies of Johnson & Johnson
- Daniel Chung - Chief Medical Officer, Sparing Vision
4:15pm - 5:00pm45 mins
General Session
Breaking Down the Four Identified Rare Disease Landscapes
- Casey McPherson - Founder and President, To Cure A Rose Foundation
- Wendy Erler - VP Patient Experience, STAR, & Global Patient Advocacy, Alexion Pharmaceuticals: Astrazeneca Rare Disease
- Chris Garabedian - CEO, Xontogeny LLC
5:00pm - 6:00pm60 mins
Close of Day and Networking Reception