RARE DISEASE DAY 2019

In recognition of Rare Disease Day, Rocket Pharmaceuticals Inc., in collaboration with the Alexandria Center for Life Science, hosted its first Rare Disease Day NYC event at the Alexandria Center for Life Science on Thursday, February 28, 2019. This patient-centered event featured patients and families sharing their personal journeys with the NYC biotech community. Capping this moving event, where speakers shared stories of living with Fanconi anemia, Danon disease, X-linked agammaglobulinemia, and spinal muscular atrophy, was the lighting of the Empire State Building in the colors commemorating Rare Disease Day.

Rare Disease Day 2019 Video Presentation

Welcoming Remarks Part 1
Gaurav Shah, M.D., CEO of Rocket Pharma

Welcoming Remarks Part 2 Jenna Foger– SVP, Science & Technology, Alexandria Venture Investments/Alexandria Real Estate Equities

What is Rare Disease Day and why do we commemorate it?
Part 1
Kinnari Patel, PharmD, MBA– COO & EVP, Development

What is Rare Disease Day and why do we commemorate it?
Part 2
Gayatri Rao, MD, JD– VP, Regulatory Policy & Patient Advocacy

What is Rare Disease Day and why do we commemorate it?
Part 3
Gayatri Rao, MD, JD– VP, Regulatory Policy & Patient Advocacy

Patient and Family Journeys Introduction
Kinnari Patel, PharmD, MBA– COO & EVP, Development

Patient and Family Journeys
Part 1
Adam Becker– A perspective on Fanconi Anemia

Patient and Family Journeys
Part 2
Adam Becker– A perspective on Fanconi Anemia

Patient and Family Journeys
Part 3
Adam Becker– A perspective on Fanconi Anemia

Patient and Family Journeys
Part 4
Adam Becker– A perspective on Fanconi Anemia

Patient and Family Journeys
Part 5
Adam Becker– A perspective on Fanconi Anemia

Patient and Family Journeys
Part 1
John Boyle – a perspective on X-linked Agammaglobulinemia

Patient and Family Journeys
Part 2
John Boyle – a perspective on X-linked Agammaglobulinemia

Patient and Family Journeys
Part 3
John Boyle – a perspective on X-linked Agammaglobulinemia

Patient and Family Journeys
Part 4
John Boyle – a perspective on X-linked Agammaglobulinemia

Patient and Family Journeys
Part 5
John Boyle – a perspective on X-linked Agammaglobulinemia

Patient and Family Journeys
Part 6
John Boyle – a perspective on X-linked Agammaglobulinemia

Patient and Family Journeys
Part 7
John Boyle – a perspective on X-linked Agammaglobulinemia

Patient and Family Journeys
Part 1
Kim Marincik – A perspective on Danon Disease

Patient and Family Journeys
Part 2
Kim Marincik – A perspective on Danon Disease

Patient and Family Journeys
Part 3
Kim Marincik – A perspective on Danon Disease

Patient and Family Journeys
Part 4
Kim Marincik – A perspective on Danon Disease

Patient and Family Journeys
Part 5
Kim Marincik – A perspective on Danon Disease

Patient and Family Journeys
Part 6
Kim Marincik – A perspective on Danon Disease

Patient and Family Journeys
Part 7
Kim Marincik – A perspective on Danon Disease

Introduction to Eddie Ndopu
Kinnari Patel, PharmD, MBA– COO & EVP, Development

Eddie Ndopu
A perspective on Spinal Muscular Atrophy (Recorded message)

Panel Discussion
Part 1
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Panel Discussion
Part 2
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Panel Discussion
Part 3
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Panel Discussion
Part 4
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Panel Discussion
Part 5
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Panel Discussion
Part 6
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Panel Discussion
Part 7
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Panel Discussion
Part 8
Adam Becker, John Boyle, Kim Marincik, Andre Hessels

Closing Remarks
Roderick Wong, MD – Managing Partner, RTW Investments

Closing Remarks
Kinnari Patel, PharmD, MBA– COO & EVP, Development

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