Approximately 10% of the American population is grappling with rare diseases, and on a global scale, over 300 million people are affected. Unfortunately, many of these individuals and their families find themselves in isolation, seeking answers to their medical inquiries. Fortunately, there's an opportunity to change this narrative.

Rare Disease Day serves as a worldwide effort to increase awareness and garner support for those navigating the challenges of rare medical conditions. Mark your calendar for the last day of February, particularly this year – February 29, the rarest day of the year – when we unite to make a difference.

Rocket Pharma is dedicated to developing best-in-class curative gene therapies for rare disorders. As part of that mission, each year Rocket organizes a Rare Disease Day event that honors the patients, families and caregivers impacted by rare diseases, highlights promising research, and shares perspectives from varied stakeholders across the rare disease community.

For Rare Disease Day 2024, Rocket will host a live and virtual event on Thursday, February 29, dedicated to improving understanding of the rare disease patient journey from diagnosis to clinical trial participation.

Rocket invites all members of the rare disease community to join the live webcast of this event. The event is free. Registration is required.
Livestream Webinar 
Date & Time
February 29, 2024,
10AM - 4PM EST
10:00 AM
Welcome | The Meaning of Rare Disease Day
Kinnari Patel, PharmD, MBA, President & Chief Operating Officer, Rocket Pharma
10:15 AM
Event Overview | Leap into Action for Rare
Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma
10:25 AM
Disease Overview | Dilated Cardiomyopathy (DCM)
Matthew Coggins, MD, Senior Medical Director, AAV, Rocket Pharma
10:30 AM
Panel Discussion | Rare Disease Diagnosis Odyssey
Did you know that up to 50% of cardiomyopathy cases are due to genetic causes but only 1% of patients get genetically tested? Genetic testing is available but not widely utilized. Hear from a patient with BAG3-associated dilated cardiomyopathy (DCM), which is a rare, genetically driven form of heart failure, a patient advocate, and a genetic counselor on why that can impact families, how to get genetic testing, and where to look for resources.

  • Panelists:
Becky, DCM 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
11:20 AM
Tribute | Remembering Vicki Modell
As co-founder of the Jeffrey Modell Foundation and a dedicated advocate for patients with primary Immunodeficiency diseases, Vicki Modell touched the lives of millions worldwide.  Her legacy endures.

Presented by Gayatri Rao, MD, JD, Chief Development Officer, LVV, Rocket Pharma
11:30 AM
Lunch Break
1:00 PM
Disease Overview | Amyloidosis
Eileen Nicoletti, MD, Medical Director, Fanconi Anemia/PKD, Rocket Pharma
1:05 PM
The Man with Nine Lives
Meet a patient living with a rare genetic amyloidosis – a disorder that occurs when amyloid proteins build up in tissues and organs – who has lived through heart and bone marrow transplants and is in remission from multiple myeloma. Be inspired by his resilience in facing life's challenges including the compromising and life-threatening side effects of the medicines, risk of secondary diseases and countless nights in hospital beds and be uplifted by his hope and determination to treat each day as a miracle.

  • Altaf, Rare Amyloidosis Patient, Biotech/Biopharma Commercial Consultant and Leader
  • Moderator: Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma
1:35 PM
Mindfulness Meditation and Rare Disease:  Taking Care of Your Mind
Welcome a special guest to hear how meditation can help patients and families affected by rare diseases to reduce stress, manage anxiety and enhance mental health.

  • David Nichtern, Emmy Award-Winning Composer & Musician and Buddhist Teacher
  • Introduction: Jonathan Schwartz, MD, Chief Gene Therapy Officer, Rocket Pharma
2:25 PM
2:40 PM
Forbes Spotlight | Inside Rocket Pharma’s Pursuit of Gene Therapy Cures for Rare Diseases
This panel will offer perspective from Rocket Pharma executives about the company’s pioneering efforts in rare disease research, emphasizing the critical role of the global patient community, sharing stories about overcoming challenges, and discussing the future of gene therapy.

  • Panelists:
Maneet Ahuja, Editor-At-Large, Forbes  
Gaurav Shah, MD, Chief Executive Officer, Rocket Pharma
Kinnari Patel, PharmD, MBA, President & Chief Operating Officer, Rocket Pharma
3:10 PM
Closing Remarks
Gaurav Shah, MD, Chief Executive Officer, Rocket Pharma
3:15 PM
Adjournment of Livestream
DCM Patient and Caregiver
Greg Ruf
Executive Director &
Board President
DCM Foundation
Abigail Yesso, MS
Genetic Counselor
Texas Childrens
Rare Amyloidosis Patient
Biotech/Biopharma Commercial Consultant and Leader
David Nichtern
Emmy Award-Winning Composer & Musician and Buddhist Teacher
Maneet Ahuja
Gaurav Shah, MD
Chief Executive Officer
Rocket Pharma
Kinnari Patel, PharmD, MBA
President & Chief Operating Officer
Rocket Pharma
Matthew Coggins, MD
Senior Medical Director, AAV
Rocket Pharma
Latika Hickey, PharmD
Senior Director, Patient Advocacy
Rocket Pharma
Eileen Nicoletti, MD
Medical Director,
 Fanconi Anemia/PKD
Rocket Pharma
Gayatri Rao MD, JD
Chief Development Officer, LVV
Rocket Pharma
Jonathan Schwartz, MD
Chief Gene Therapy Officer
Rocket Pharma
Erika Zannou-Mantri PharmD, PhD and MBA
SVP, Development Operations & Global Program Head
Rocket Pharma
Thank you to our 2024 Rare Disease Day Sponsors
Rare Resilience Partner
Striped Supporter
In Grateful Appreciation
Rod & Marti Speranza Wong
 Join us online on February 29 
We look forward to you joining
Rocket Pharma's Rare Disease Day Event

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