SPEAKERS FROM ALL OVER THE WORLD

 

 

 

Ms Karen Aiach, Chief Executive Officer, Lysogene

Einar Andreassen, Senior Adviser, Norwegian Medicines Agency

Diego Ardigo, Project Leader for Advanced Therapies, CHIESI Farmaceutici Spa

Diego Ardigo at World Orphan Drug Congress USA 2017

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

Alison Bateman House, Assistant Professor, Division of Medical Ethics, New York University Langone Medical Center

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

Ms Michelle Berg, Vice President, Patient Advocacy, Abeona Therapeutics Inc.

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

John Boland, Vice President of Product Development, Atlantic Research Group Inc

John Boland at World Orphan Drug Congress USA 2017

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

Molly Burich, Associate Director of Public Policy, Biosimilars, Pipeline and Reimbursement, Boehringer Ingelheim

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

Carlos Camozzi, Group Chief Medical Officer, Simbec-Orion Group

Jeff Ceitlin, Chief Commercial Officer, HVH Patient Precision Analytics

Angela Chaves, President, FEDERACION COLOMBIANA DE ENFERMEDADES RARAS

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

Jodi Cook, Chief Operating Officer, Agilis Biotherapeutics

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

John F. Crowley, Chairman and Chief Executive Officer, Amicus Therapeutics

John F. Crowley at World Orphan Drug Congress USA 2017

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

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

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

Wildon Farwell, Senior Medical Director, Clinical Development, Biogen

Wildon Farwell at World Orphan Drug Congress USA 2017

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

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

Jayne Gershkowitz, 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

Gudrun Helga Haroardottir, Director, Einstok Born Support Group For Children With Rare Disorders

Gudrun Helga Haroardottir 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

Michelle Hylan, ‎Innov8tive Research Consulting, Allena Pharmaceuticals Inc

Michelle Hylan at World Orphan Drug Congress USA 2017

Raul Insa, Founder and Chief Executive Officer, SOM Biotech SL

Raul Insa at World Orphan Drug Congress USA 2017

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

Matt Kapusta, Chief Executive Officer, uniQure

Matt Kapusta at World Orphan Drug Congress USA 2017

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

Takashi K Kishimoto, Chief Scientific Officer, Selecta Biosciences Inc

Ragnar Klingberg, CEO, EmeraMed

Christopher Komelasky, Senior Director, Site and Patient Access, PPD

Walter Kowtoniuk, Director, Strategy and Operations, Fulcrum Therapeutics

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

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

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

Alastair Macdonald, Exec Director, RWLP Strategy, Global, INC Research

Alastair Macdonald 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

John Maslowski, CEO, Fibrocell

John Maslowski at World Orphan Drug Congress USA 2017

Mr Gary Mathias, Chief Executive Officer, Thetis Pharmaceuticals Llc

Mr Gary Mathias at World Orphan Drug Congress USA 2017

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

Mr 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

Juliet Moritz, Executive Director, Strategic Development, Rare Diseases, Premier Research

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

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

Joseph Musumeci, Senior Advisor, BluePrint Orphan

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, President, NPO, 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

Anne Pariser, Deputy Director, Office of Rare Diseases Research, NCATS, NIH

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

Horacio Plotkin, VP, Global Therapeutic Head, Pediatrics & Rare Diseases, PPD

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, CADTH

Mark Rothera, Chief Commercial Officer, PTC Therapeutics

Mark Rothera at World Orphan Drug Congress USA 2017

Peter Saltonstall, CEO, National Organization For Rare Disorders

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

Bhavneesh Sharma, Founder, Managing Principal and Portfolio Manager, Vasuda Capital Managament

Alex Sherman, Director of Strategic Development and Systems, Massachusetts General Hospital Neurological Clinical Research Institute

Sascha Sonnenberg, Dipl.-Oec., MBA, Vice President Global CTD Sales & Operations, Marken

Sascha Sonnenberg at World Orphan Drug Congress USA 2017

Mark Sorrentino, Vice President, Scientific Affairs - Pediatrics, PRA Health Sciences

Mark Sorrentino at World Orphan Drug Congress USA 2017

Barbara Sosnowski, VP of E.R.D.I. and Head of Cambridge Research Units, Pfizer, Inc

Barbara Sosnowski at World Orphan Drug Congress USA 2017

Jeremy Springhorn, Partner Corporate Development, Flagship Ventures

Thomas Sproat, Senior Vice President, Scientific Services, ClinicalMind

Thomas Sproat at World Orphan Drug Congress USA 2017

Stephen Squinto, Venture Partner, Orbimed

Jamie Topper, Managing General Partner, Frazier Healthcare Ventures

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

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

Julie Walters, Founder, Raremark

Julie Walters at World Orphan Drug Congress USA 2017

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

Michael Wyand, Former President and Chief Operating Officer, EPIRUS Biopharma

Michael Wyand at World Orphan Drug Congress USA 2017

Chen Yu, Managing Partner, Vivo Capital

 

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