SPEAKERS FROM ALL OVER THE WORLD

 

 

 

Karen Aiach, Founder and Chief Executive Officer, Lysogene

Einar Andreassen, Senior Adviser, Norwegian Medicines Agency

Donna Armentano, Global Head of Gene Therapy, Pfizer, Inc

Alison Bateman House, Postdoctoral Fellow, Division of Medical Ethics,, New York University Langone Medical Center

Ms Soraya Bekkali, Senior Vice President and Chief Medical Officer, Lysogene

Stella Blackburn, VP, Global Head of Risk Management, QuintilesIMS

Stella Blackburn at World Orphan Drug Congress USA 2017

Dale Bodian, Manager Bioinformatics Scientist, Inova Translational Medicine Institute

Katrine Bosley, Chief Executive Officer, Editas Medicine

Meriem Bouslouk, Officer, Innovation Committee, Federal Joint Committee

Murray Brilliant, Senior Research Scientist and Center Director, Marshfield Clinic Research Foundation

Max Bronstein, Senior Director, Public and Government Relations, EveryLife Foundation for Rare Diseases

Anna Bucsics, External Lecturer, MoCA board member, University of Vienna

Anna Bucsics at World Orphan Drug Congress USA 2017

Andrea Califano, Chair, Systems Biology Department, Columbia University U.S.A.

Jeff Ceitlin, Chief Commercial Officer, HVH Patient Precision Analytics

Jeff Ceitlin at World Orphan Drug Congress USA 2017

Angela Chaves, President, FEDERACION COLOMBIANA DE ENFERMEDADES RARAS

Andrea Chiesi, Director of R&D and Portfolio Management, Chiesi

William W. Chin, CMO, Executive VP, Science and Regulatory Affairs, PhRMA

Leah Christl, AD,Therapeutic Biologics & Biosimilars, F.D.A.

Leah Christl at World Orphan Drug Congress USA 2017

Margaret Collins, Professor of Pediatrics, Cincinnati Childrens Hospital

Francesca Cook, Director, Pricing and Market Access, RegenxBio

Francesca Cook at World Orphan Drug Congress USA 2017

Invited: John Coster, Director, Division of Pharmacy, Center for Medicare and Medicaid Services

Gregory Daniel, Deputy Director, Duke University

Mark de Boer, Chief Executive Officer, FF Pharma

Peter Dehnel, Medical Director, Blue Cross and Blue Shield of Minnesota

Peter Dehnel at World Orphan Drug Congress USA 2017

Keith DeMatteo, Director, Global Procurement, Bristol Myers Squibb

Kelly Du Plessis, Chief Executive Officer, Rare Disease Society of South Africa

Kelly Du Plessis at World Orphan Drug Congress USA 2017

Valerie Duquet, Clinical Team Manager, PRA Health Sciences

Valerie Duquet at World Orphan Drug Congress USA 2017

Paul Eccles, PhD, Client Services Director, Cello Health Insight

Glyn Edwards, Chief Executive Officer, Summit Therapeutics

Florian Eichler, Associate Professor of Neurology, Massachusetts General Hospital

David Fajgenbaum, Assistant Professor of Medicine, Upenn Center For Orphan Disease Research and Therapy

Thomas J Farrell, Chief Executive Officer, Bellicum Pharmaceuticals, Inc.

Mary Frances Harmon, Head of Global Patient Advocacy, PTC Therapeutics

Mary Frances Harmon at World Orphan Drug Congress USA 2017

Pat Furlong, Founding President and Chief Executive Officer, Parent Project Muscular Dystrophy

William Gahl, Clinical Director, Director of the N.I.H. Undiagnosed Diseases Program

Vignesh Ganapathy, AD of Advocacy & Government Relations, EveryLife Foundation for Rare Diseases

Vignesh Ganapathy at World Orphan Drug Congress USA 2017

Chris Garabedian, Former President and Chief Executive Officer, Sarepta Therapeutics

Eric Gascho, Vice President of Government Affairs, National Health Council

Deborah Geraghty, Head of Commercial and Portfolio Strategy, Dimension Therapeutics

Jayne Gershkowitz, Senior Vice President and Chief Patient Advocate, Amicus Therapeutics Inc

Michael Goettler, Global President, Pfizer Rare Disease

Jonathan Goldsmith, Associate Director Rare Diseases Program, Food And Drug Administration

Jonathan Goldsmith at World Orphan Drug Congress USA 2017

Rashmi Gopal-Srivastava, Dir, Extramural Research Program, Office Rare Diseases, National Institutes of Health

Hazel Gorham, Director of Biosimilars Development, P.R.A. Health Sciences

Hazel Gorham at World Orphan Drug Congress USA 2017

Scott Gray, Chief Executive Officer, Clincierge

James Greenwood, Chief Executive Officer, Biotechnology Industry Organization

Yi Han, PhD, MBA, Executive Vice President of Market Access and HEOR, Cello Health Communications

Colin Hayward, Chief Medical Officer, Premier Research

Jonathan Heller, Global Head, External R&D Innovation,Rare Diseases, Pfizer, Inc

Jonathan Heller at World Orphan Drug Congress USA 2017

Johan Heylen, Chief Commercial Officer, Ablynx

Claudia Hirawat, Chair, EURORDIS International Circle of Ambassadors

Kenneth Hobby, President, Cure SMA

Angus Hogg, Vice President Hunters (MPSII) Franchise, Shire Pharmaceuticals Ltd

Angus Hogg at World Orphan Drug Congress USA 2017

Kay holcombe, Senior Vice President for Science Policy, Biotechnology Industry Organization (BIO)

Kay holcombe at World Orphan Drug Congress USA 2017

Wills Hughes-Wilson, Senior Vice President, Chief Patient Access Officer, Sobi

Dr Cynthia Jackson, VP & Head, Pediatric & Rare Disease Cnrts of Excellence, QuintilesIMS

Dr Cynthia Jackson at World Orphan Drug Congress USA 2017

Edmund Jessop, Medical Adviser, National Health Service

Donald Johns, VP, Head of ALS and SMA Clinical Development, Biogen

Matt Kapusta, Chief Executive Officer, uniQure

Matt Kapusta at World Orphan Drug Congress USA 2017

Petra Kaufmann, Director, Office of Rare Diseases Research, National Institutes of Health

Sven Kili, Vice President and Head of Gene Therapy Development, GSK

Takashi K Kishimoto, Chief Scientific Officer, Selecta Biosciences Inc

Jennifer Kwon, Associate Professor of Neurology and Pediatrics, University of Rochester

Mr S.H. Lau, CEO, New Biotic, LLC

Mr S.H. Lau at World Orphan Drug Congress USA 2017

Yann Le Cam, Chief Executive Officer, EURORDIS

Nadea Leavitt, Engagement Mgr, Predictive Analytics, RW Insights, QuintilesIMS

Nadea Leavitt at World Orphan Drug Congress USA 2017

Mr John Leonard, CMO, Intellia Therapeutics Inc

Nick Leschly, President and Chief Executive Officer, bluebird bio

Terrie Livingston, Senior Director, US Medical, Biogen

Aoife M Brennan, Chief Medical Officer, Synlogic Therapeutics

Aoife M Brennan at World Orphan Drug Congress USA 2017

Mr Stephen Marcus, Chief Executive Officer, Cantex Pharmaceuticals

Jeffrey D. Marrazzo, Co Founder and Chief Executive Officer, Spark Therapeutics

Marc Martinell, Chief Executive Officer, Minoryx Therapeutics

Howard Mayer, SVP & Head of Global Clinical Development, Shire Pharmaceuticals Inc

James McDermott, Vice President, Cello Health BioConsulting

Andrew McFayden, Executive Director, The Isaac Foundation

Andrew McFayden at World Orphan Drug Congress USA 2017

Paul Melmeyer, Associate Director of Public Policy, National Organization for Rare Disorders

Paul Melmeyer at World Orphan Drug Congress USA 2017

Jerry R. Mendell, Director, Center for Gene Therapy, Nationwide Children's Hospital

Christopher Missling, President and Chief Executive Officer, Anavex Life Sciences

Christopher Missling at World Orphan Drug Congress USA 2017

Kenneth Moch, President & CEO, Cognition Therapeutics; Former President & CEO, Chimerix

Richard Moscicki, Deputy Director for Science Operations, CDER, FDA

Michael Murphy, Chief Medical & Scientific Officer, Worldwide Clinical Trials

Jeff Myers, CEO, Medicaid Health Plans of America

Francois Nader, Former President & CEO, NPS, Board Chairman, Acceleron Pharma

Nikhil Nair, Assist Professor, Dept of Chemical and Biological Engineering, Tufts University

Yukiko Nishimura, Chief Secretariat of International Relations, Japan Patients Association, ASrid

Letitia O'dwyer, Chief Executive Officer, New Zealand Organisation For Rare Disorders

Ethan Oren Perlstein, Chief Executive Officer, Perlstein Labs

Romina Ortiz, VP Policy & Patient Advocacy, Rare Genomics Institute

Luca Pani, Former Gen Director AIFA, CHMP and SAWP Member, European Medicines Agency

Detlev Parow, Head, Care Management Development, D.A.K.

Nancy Parsons, VP Commercial Operations, Pharma Products, SigmaTau Pharmaceuticals

Nancy Parsons at World Orphan Drug Congress USA 2017

Eric Pauwels, SVP & General Manager, Americas, PTC Therapeutics

Edmund Pezalla, Fomer VP Pharmaceutical Policy and Strategy, Aetna

Andreas Pleil, Sr Dr, Team Lead, Outcomes & Evidence Hematology, Pfizer Inc

Sy Pretorius, SVP and Chief Scientific Officer, PAREXEL International

Harsha Rajasimha, Co Founder, Organization for Rare Diseases India

Gayatri Rao JD., MD,, Director, Orphan Products Development, FDA

Adam Resnick, Director, Ctr for Data Driven Discovery in Biomedicine, The Children's Hospital of Philadelphia

Trevor Richter, Director, Common Drug Review and Optimal Use, C.A.D.T.H.

Scott Rocklage, Frmer CEO, Epirus Biosciences, Partner, 5AM Ventures

Scott Rocklage at World Orphan Drug Congress USA 2017

Mark Rothera, Chief Commercial Officer, PTC Therapeutics

Mark Rothera at World Orphan Drug Congress USA 2017

Peter Saltonstall, CEO, National Organization For Rare Disorders

Ms Amber Salzman, President and Chief Executive Officer, Adverum Biotechnologies

Amanda Samanek, Executive Director, Genetic and Rare Disease Network, Australia

Amanda Samanek at World Orphan Drug Congress USA 2017

Scott Schliebner, VP, Rare Diseases & Scientific Affairs, PRA Health Sciences

Professor Matthias Schönermark, Chief Executive Officer, SKC Beratungsgesellschaft

Professor Matthias Schönermark at World Orphan Drug Congress USA 2017

Ana Maria Serrato Navas, Country Manager, Colombia, Aegerion Pharmaceuticals

Ana Maria Serrato Navas at World Orphan Drug Congress USA 2017

Samuel Seward, Site Chair, Department of Medicine, Mount Sinai Hospital

Samuel Seward at World Orphan Drug Congress USA 2017

Thomas Sproat, Senior Vice President, Scientific Services, ClinicalMind

Thomas Sproat at World Orphan Drug Congress USA 2017

Mary Szela, Chief Executive Officer, Aegerion Pharmaceuticals

Birgitte Volck, Senior Vice President, Head of R&D Rare Diseases, GSK

Timothy Walbert, Chairman, President, and Chief Executive Officer, Horizon Pharma

Diane White, Assoc. Dir Development Sciences, Biomarin

Diane White at World Orphan Drug Congress USA 2017

James Wilson, Director, Orphan Disease Center, University of Pennsylvania

Marc Wolff, EVP & CFO, Sancilio Pharmaceuticals Company

Barbara Wuebbels, Vice President of Patient Advocacy, Audentes Therapeutics

 

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Contact Andre Singer at andre.singer@terrapinn.com
or  +1 646 619 1797