RARE DISEASE DAY 2024
ROCKET PHARMA CELEBRATES RARE DISEASE DAY 2024
LEAP INTO ACTION FOR RARE
At Rocket, we are laser-focused on addressing the unmet needs of patients living with rare, life-threatening diseases. We understand living with a rare disease is much more than the disease itself. It involves fighting misconceptions, finding informed, patient and compassionate caregivers, and seeking accurate information about available treatment options.
As ardent advocates for the rare disease community, Rare Disease Day presents a timely opportunity to raise awareness and contribute to educating communities at the highest level, while aiming to ensure patients and their loved ones have access to the information and resources that they need to help connect and empower them on their journey.
In celebration of Rare Disease Day 2024, Rocket held an event at the Liberty Science Center in Jersey City, NJ. Among several topics, the event featured a tribute to Vicki Modell, cofounder of the Jeffery Modell Foundation, and inspiring advocate for patients living with primary immunodeficiency diseases, and inspirational stories from patients living with rare diseases.
Welcome & Event Overview l The Meaning of Rare Disease Day
Opening Remarks: Kinnari Patel, PharmD, MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma
Overview of Event: Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma
Panel Discussion l Rare Disease Diagnosis Odyssey
Disease Overview: Matthew Coggins, MD, Senior Medical Director, AAV, Rocket Pharma
Panelists:
Becky, Dilated Cardiomyopathy Patient and Caregiver
Abigail Yesso, MS, Genetic Counselor, Texas Childrens
Greg Ruf, Executive Director & Board President, DCM Foundation
Moderator: Erika Zannou-Mantri, PharmD, PhD, MBA, SVP, Development Operations & Global Program Head, Rocket Pharma
Tribute l Remembering Vicki Modell
Gayatri Rao, MD, JD, Chief Regulatory Officer & SVP, Clinical Safety, Rocket Pharma
The Man with Nine Lives
Disease Overview: Eileen Nicoletti, MD, Medical Director, Fanconi Anemia/PKD, Rocket Pharma
Panelist: Altaf, Rare Amyloidosis Patient, Biotech/Biopharma Commercial Consultant and Leader
Moderator: Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma
Mindfulness Meditation and Rare Disease: Taking Care of Your Mind
Introduction: Jonathan Schwartz, MD, Chief Medical & Gene Therapy Officer, Rocket Pharma
Speaker: David Nichtern, Emmy Award-Winning Composer & Musician and Buddhist Teacher
Forbes Spotlight l Inside Rocket Pharma’s Pursuit of Gene Therapy Cures for Rare Diseases
Panelists:
Maneet Ahuja, Editor-At-Large, Forbes
Gaurav Shah, MD, Chief Executive Officer, Rocket Pharma
Kinnari Patel, PharmD, MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma
