RARE DISEASE DAY 2019
Rare Disease Day 2019 Video Presentation
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Welcoming Remarks Part 1
Gaurav Shah, M.D., CEO of Rocket Pharma
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What is Rare Disease Day and why do we commemorate it?
Part 1
Kinnari Patel, PharmD, MBA– COO & EVP, Development
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What is Rare Disease Day and why do we commemorate it?
Part 2
Gayatri Rao, MD, JD– VP, Regulatory Policy & Patient Advocacy
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What is Rare Disease Day and why do we commemorate it?
Part 3
Gayatri Rao, MD, JD– VP, Regulatory Policy & Patient Advocacy
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Patient and Family Journeys Introduction
Kinnari Patel, PharmD, MBA– COO & EVP, Development
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Patient and Family Journeys
Part 1
Adam Becker– A perspective on Fanconi Anemia
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Patient and Family Journeys
Part 2
Adam Becker– A perspective on Fanconi Anemia
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Patient and Family Journeys
Part 3
Adam Becker– A perspective on Fanconi Anemia
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Patient and Family Journeys
Part 4
Adam Becker– A perspective on Fanconi Anemia
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Patient and Family Journeys
Part 5
Adam Becker– A perspective on Fanconi Anemia
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Patient and Family Journeys
Part 1
John Boyle – a perspective on X-linked Agammaglobulinemia
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Patient and Family Journeys
Part 2
John Boyle – a perspective on X-linked Agammaglobulinemia
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Patient and Family Journeys
Part 3
John Boyle – a perspective on X-linked Agammaglobulinemia
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Patient and Family Journeys
Part 4
John Boyle – a perspective on X-linked Agammaglobulinemia
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Patient and Family Journeys
Part 5
John Boyle – a perspective on X-linked Agammaglobulinemia
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Patient and Family Journeys
Part 6
John Boyle – a perspective on X-linked Agammaglobulinemia
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Patient and Family Journeys
Part 7
John Boyle – a perspective on X-linked Agammaglobulinemia
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Patient and Family Journeys
Part 1
Kim Marincik – A perspective on Danon Disease
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Patient and Family Journeys
Part 2
Kim Marincik – A perspective on Danon Disease
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Patient and Family Journeys
Part 3
Kim Marincik – A perspective on Danon Disease
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Patient and Family Journeys
Part 4
Kim Marincik – A perspective on Danon Disease
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Patient and Family Journeys
Part 5
Kim Marincik – A perspective on Danon Disease
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Patient and Family Journeys
Part 6
Kim Marincik – A perspective on Danon Disease
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Patient and Family Journeys
Part 7
Kim Marincik – A perspective on Danon Disease
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Introduction to Eddie Ndopu
Kinnari Patel, PharmD, MBA– COO & EVP, Development
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Eddie Ndopu
A perspective on Spinal Muscular Atrophy (Recorded message)
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Panel Discussion
Part 1
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Panel Discussion
Part 2
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Panel Discussion
Part 3
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Panel Discussion
Part 4
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Panel Discussion
Part 5
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Panel Discussion
Part 6
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Panel Discussion
Part 7
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Panel Discussion
Part 8
Adam Becker, John Boyle, Kim Marincik, Andre Hessels
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Closing Remarks
Roderick Wong, MD – Managing Partner, RTW Investments
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Closing Remarks
Kinnari Patel, PharmD, MBA– COO & EVP, Development