RARE DISEASE DAY 2022
ROCKET PHARMA CELEBRATES RARE DISEASE DAY 2022
Rare, but Not Alone
Our mission is to seek gene therapy cures for people living with rare diseases, who are our North Star. During Rocket’s Rare Disease Day 2022 celebration, 250+ members of the rare disease community and Rocket team gathered at the NASDAQ Tower in Times Square and virtually.
Introduction to Rare Disease Day
Kinnari Patel, PharmD, MBA
Danon Disease & Multi-Generational Care: A Rare Patient and Family Story
Moderator: Latika Hickey, PharmD
Speaker Panel: Dawn, Barbara, Steven, and Gabriel
Rare Innovations: Harnessing Collaboration to Drive Medical Innovation in DanonDisease
Moderator: Gaurav Shah, MD
Keynote Speaker: MorisDanon, MD
Speaker Panel: Matthew Taylor, MD, PhD; Eric Adler, MD; Barry Greenberg, MD; Joseph Rossano, MD
Rare Resources
RTW Foundation: Deborah Slipetz, PhD
DanonFoundation: Kim Marincik, Jenny Hsieh, Amy Atkinson
National Organization for Rare Disorders (NORD): Debbie Drell
Rare, But Not Alone
Mousumi Bose, PhD
Danielle Kinsey, MD
Lucas and Emma (PKD)
Paul and Barbara (IMO)
Shaping Foundations
About Shaping Foundations
“I Support Rare”
Closing Remarks & Musical Performance
Gaurav Shah, Falu, Sarosh Effendi
