RARE DISEASE DAY 2023
ROCKET PHARMA CELEBRATES RARE DISEASE DAY 2023
STORIES THAT NEED TO BE SHARED: THE HUMAN SIDE OF RARE DISEASE
Our mission is to seek gene therapy cures for people living with rare diseases, who are our North Star. In celebration of Rare Disease Day 2023, Rocket held an event at the Make-A-Wish New Jersey Samuel & Josephine Plumeri Wishing Place in Monroe Township, NJ.
Resembling a fairy tale castle and dedicated to granting the wishes of children with critical illnesses, the Make-A-Wish venue provided a meaningful backdrop to the event that brought together more than 200 people and featured inspirational stories from the global rare disease community.
Welcoming Remarks
Kinnari Patel, PharmD, MBA, President & Chief Operating Officer, Rocket Pharma
Event Overview: Stories that Need to Be Shared: The Human Side of Rare Diseases
Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma
Ensuring Patient Access to Rare Disease Treatments
Claire Booth, MBBS PhD, Prof. Gene Therapy and Pediatric Immunology, University College of London (UCL), Great Ormond Street Institute of Child Health, Co-founder, AGORA (Access to Gene Therapies for Rare Disease)
Patient & Caregiver Perspective: Arrhythmogenic Cardiomyopathy (ACM)
Moderator: Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma
Speaker Panel: Maddie and Michelle
Raising Awareness of Sudden Arrhythmia Death Syndromes (SADS)
Alice Lara, RN, President and CEO, SADS Foundation
Patient & Caregiver Perspective: Pyruvate Kinase Deficiency (PKD)
Moderator: Gayatri Rao, M.D., J.D., Chief Development Officer, LVV, Rocket Pharma
Speaker Panel: Travis and Jessica
Patient Advocacy Video Resources
“Happy Rare Disease Day” from Shaping Foundations
Thrive with Pyruvate Kinase Deficiency (PKD)
Pyruvate Kinase Deficiency Foundation (PKDF)
Closing Remarks
Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma
