Main Day 1

9:00 AM - 9:45 AM Defining PAPs with the Future in Mind: Gene Therapy and What’s to Come

Pat Furlong, President & CEO, Parent Project Muscular Dystrophy

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Pat Furlong

President & CEO
Parent Project Muscular Dystrophy

10:00 AM - 10:45 AM Weighing your Options: Considering, Selecting and Managing a PAP Effectively

Catherine Blansfield, Vice President of Patient Services , National Organization for Rare Disorders (NORD)

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Catherine Blansfield

Vice President of Patient Services
National Organization for Rare Disorders (NORD)

11:30 AM - 12:15 PM The Moral Dilemma: Providing Potentially Life-Saving Drugs in an Ethical Manner

Andrew McFadyen, Executive Director, The Isaac Foundation

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Andrew McFadyen

Executive Director
The Isaac Foundation

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Brian Malkin

FDA Counsel
Arent Fox LLP
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Tom Watson

Pre-approval Access Consultant
TW Consulting Group

Andrew McFadyen

Executive Director
The Isaac Foundation

Luiz Andre Magno

Global Medical Affairs Leader - Neuroscience
Janssen Pharmaceutical

1:45 PM - 2:30 PM The Expanded Access (EA) Navigator Program for Single Patient or Compassionate Use: A One Stop Shop

June Wasser, Executive Director and CEO, Reagan-Udall Foundation for the FDA

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June Wasser

Executive Director and CEO
Reagan-Udall Foundation for the FDA

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Paul Melmeyer

Director of Federal Policy
National Organization for Rare Disorders (NORD)
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Alison Bateman-House

Assistant Professor – Division of Medical Ethics
NYU
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Brian Malkin

FDA Counsel
Arent Fox LLP
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Naomi Lopez Bauman

Director of Healthcare Policy
Goldwater Institute

3:45 PM - 4:30 PM The Considerations Taken Into Account at to Build a Successful PAP

Karen Bartels, Director, Global Medical Affairs, Early Clinical Development and External Research , AstraZeneca

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Karen Bartels

Director, Global Medical Affairs, Early Clinical Development and External Research
AstraZeneca

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Naomi Lopez Bauman

Director of Healthcare Policy
Goldwater Institute
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Frank Burroughs

CEO
Abigail Alliance for Better Access to Developmental Drugs

Kevin J. Weatherwax

Administrative Program Director; Co-Chair - Expanded Access Oversight Committee
University of Michigan Health System

Karen Bartels

Director, Global Medical Affairs, Early Clinical Development and External Research
AstraZeneca