RARE DISEASE DAY 2019
Rare Disease Day 2019 Video Presentation

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


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