2018 Speakers

 

Karen Aiach, Chief Executive Officer, Lysogene

Ron Alfa, Vice President of Discovery and Product, Recursion Pharmaceuticals

Christopher Anzalone, President and Chief Executive Officer, Arrowhead Research Corp

Christopher Anzalone at World Orphan Drug Congress USA 2018

Anita Appius, Global Development Team Leader, Roche

Anita Appius at World Orphan Drug Congress USA 2018

Diego Ardigo, Project Lead, Chiesi Farmaceutici SpA

Diego Ardigo at World Orphan Drug Congress USA 2018

Katri Asikainen, Chairperson, Finnish Rare Diseases and Disabilities Organization (HARSO)

Mark Baglin, Vice President of Global Marketing, Alnylam Pharmaceuticals

Mark Baglin at World Orphan Drug Congress USA 2018

Mariah Baltezegar, Executive Director Clinical Development, Rare Diseases, INC Research/inVentiv Health

Mariah Baltezegar at World Orphan Drug Congress USA 2018

Thomas Barnes, Senior Vice President, Innovative Sciences,, Intellia Therapeutics Inc.

Thomas Barnes at World Orphan Drug Congress USA 2018

Jeanne Barnett, Founder and President, Cysticfibrosis.com

Ron Bartek, Co-Founder/Founding President, Friedreich's Ataxia Research Alliance

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

Saira Bates, Co Founder and Chief Executive Officer, Escend Pharmaceuticals Inc

Denis Belyakov, CEO, Union of Patients’ And Patients’ Organizations With Rare Diseases

Carole Ben Maimon, Chief Executive Officer, Chondrial Therapeutics

Laxminarayan Bhat, Founder and Chief Executive Officer, Reviva Pharmaceuticals Inc

Dawn Bir, Chief Commercial Officer, Reata Pharmaceuticals

Hans Bishop, President and Chief Executive Officer, Juno Therapeutics

Hans Bishop at World Orphan Drug Congress USA 2018

Bruce Bloom, President, Cures Within Reach

Bruce Bloom at World Orphan Drug Congress USA 2018

David Bonita, Private Equity Partner, Orbimed Advisors

Katrine Bosley, Chief Executive Officer, Editas Medicine

Aldar Bourinbaiar, Chief Executive Officer, Immunitor Inc

Ari Brettman, Principal, Clarus Ventures

Brian Bronk, Head of External Innovation - Rare Diseases, Sanofi

Brian Bronk at World Orphan Drug Congress USA 2018

Philip J. Brooks, Program Director, Office of Rare Diseases Research, NCATS, NIH

Anne Bruns, Associate Executive Director and Operations Manager, The Atypical HUS Foundation

Anne Bruns at World Orphan Drug Congress USA 2018

Preston Campbell, President and Chief Executive Officer, Cystic Fibrosis Foundation

Rick Chapman, Director of Health Economics, Institute for Clinical and Economic Review

Angela Chavez, President, Colombia Rare Disorders Society

Angela Chavez at World Orphan Drug Congress USA 2018

Frederic Chereau, Chief Executive Officer, LogicBio Therapeutics

Frederic Chereau at World Orphan Drug Congress USA 2018

Andre Choulika, Chairman & Chief Executive Officer, Cellectis

Andre Choulika at World Orphan Drug Congress USA 2018

Daniel Cohen, Chief Executive Officer, Pharnext

Francesca Cook, Director, Pricing and Market Access, RegenxBio

Francesca Cook at World Orphan Drug Congress USA 2018

Jacqueline Corrigan-Curay, Director, Office of Medical Policy, Center for Drug Evaluation and Research, F.D.A

David Cory, President, Chief Executive Officer, Eiger Biopharmaceuticals

Gregory Daniel, Deputy Director, Duke University

Olivier Danos, Chief Scientific Officer, Regenxbio Inc

Daniel De Boer, Chief Executive Officer, Proqr

Kelly Du Plessis, Chief Executive Officer, Rare Diseases South Africa

Mark Dunning, Chairman, Coalition for Usher Syndrome Research

Mark Dunning at World Orphan Drug Congress USA 2018

David Elvira Martinez, Director of the Catalan Health Service, CatSalut, Catalunya Health Service

Susan Fenters Lerch, Executive Director, The Hemophilia Foundation of Michigan

Susan Fenters Lerch at World Orphan Drug Congress USA 2018

Beata Ferencz, Project Manager, Rare Diseases Sweden

Gregory Fond, Director, External Innovation, Rare Diseases, Sanofi

Stephen Franklin, Chief Executive, Evgen Pharma

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

Prasad Gabbita, Chief Executive Officer, P2d Inc

Pamela Gavin, Chief Operating Officer, National Organization for Rare Disorders

Jayne Gershkowitz, Chief Patient Advocate, Amicus Therapeutics

Thomas Goss, Senior Vice President, Boston Healthcare Associates Inc

Scott Gray, Chief Executive Officer, Clincierge

Peter Greenleaf, Chief Executive Officer, Sucampo Pharmaceuticals Inc

Philippe Guedat, Managing Director, In Flectis Bio Science

Rylan Hanks, Director, Regulatory and R&D Policy, Amgen Inc

Colin Hayward, Chief Medical Officer, Premier Research

Jennifer Helfer, Patient Advocacy, bluebird bio

Jennifer Helfer at World Orphan Drug Congress USA 2018

Johan Heylen, Chief Commercial Officer, Ablynx

Katherine High, President and Head, Research & Development, Spark Therapeutics

Kenneth Hobby, President, Cure SMA

Kim Hollander, Executive Director, The Oxalosis and Hyperoxaluria Foundation

Kim Hollander at World Orphan Drug Congress USA 2018

Birthe Byskov Holm, President, Rare Diseases Denmark

Bill Hornung, Chief Business Officer, Diffusion Pharmaceuticals LLC

Arnaldo Hossepian, Member, Conselho Nacional de Justiça

Arnaldo Hossepian at World Orphan Drug Congress USA 2018

Kevin Huang, President, Chinese Organization for Rare Disorders

Kevin Huang at World Orphan Drug Congress USA 2018

Adam Hutchings, Managing Director, Dolon

Douglas Ingram, Chief Executive Officer, Sarepta Therapeutics

Fred Jacobs, Chief Executive Officer and Co Founder, TYG

Edmund Jessop, Medical Adviser, National Health Service

Steven Kaminsky, Chief Science Officer, International Rett Syndrome Foundation

Karen Kaucic, Senior Vice President, Early Development, Rare Disease and Pediatric Center of Excellence, PPD® Consulting

M Ken Kengatharan, Managing Director, Atheneos Capital and Chairman, Renexxion

M Ken Kengatharan at World Orphan Drug Congress USA 2018

Alastair Kent OBE, Ambassador, Genetic Alliance U.K.

Cristina Klafehn, Associate Director,Development Sciences Patient Advocacy, BioMarin Pharmaceutical Inc.

Terri Klein, Interim President and CEO, National M.P.S. Society

Terri Klein at World Orphan Drug Congress USA 2018

Diane Kleinermans, Advisor to the Ministry of Health and Social Affairs Belgium, Belgian Federal Government

Lynnet Koh, Chairman and Chief Executive Officer and Founder, Targazyme

Suma Krishnan, Chief Operations Officer and Founder, Krystal Biotech

Suma Krishnan at World Orphan Drug Congress USA 2018

Alex La Croix, Senior Director, Rare Genetic Disease Marketing, Agios Pharmaceuticals

Alex La Croix at World Orphan Drug Congress USA 2018

David Lapidus, President, Lapidus Data

Yann Le Cam, Chief Executive Officer, EURORDIS

John Lee, Chief Medical Officer, Phasebio Pharmaceuticals, Inc.

Clement Leong, Chief Executive Officer and Managing Director, Ascend Biopharmaceuticals

Dana Levasseur, Leader and Associate Director, Bioverativ

Dana Levasseur at World Orphan Drug Congress USA 2018

Eric Li, Chief Scientific Officer, Luxena Pharmaceuticals

Edmund Lim, Co-founder, We CARE Journey

Edmund Lim at World Orphan Drug Congress USA 2018

David Litwack, Personalized Medicine Staff, U.S. Food and Drug Administration

David Litwack at World Orphan Drug Congress USA 2018

Igor Lokot, Chief Executive Officer, Double Bond Pharmaceutical A.B.

Robert Long, Director of Strategic Development, Uplifting Athletes

Robert Long at World Orphan Drug Congress USA 2018

Isabelle Lousada, CEO, Amyloidosis Research Consortium

Isabelle Lousada at World Orphan Drug Congress USA 2018

Aoife M Brennan, Chief Medical Officer, Synlogic Therapeutics

Alastair Macdonald, Exec Director, RWLP Strategy, Global, INC Research/inVentiv Health

Alastair Macdonald at World Orphan Drug Congress USA 2018

Sandy Macrae, President and Chief Executive Officer, Sangamo BioSciences, Inc

Jay Madan, President, Innovate Biopharmaceuticals Inc

Catherine Martin, Executive Director, The Huntington’s Disease Youth Organization

Catherine Martin at World Orphan Drug Congress USA 2018

Andrew McFadyen, Executive Director, The Isaac Foundation

Andrew McFadyen at World Orphan Drug Congress USA 2018

Donnie McGrath, Head of Corporate Strategy & Business Development, Biohaven Pharmaceuticals

Timothy McMahon, Professor of Pulmonary, Allergy, and Critical Care Medicine, Duke University School of Medicine

Timothy McMahon at World Orphan Drug Congress USA 2018

Meghan Miller, Project Leader/Discovery Scientist, Rare Diseases, Roche

Meghan Miller at World Orphan Drug Congress USA 2018

Tim Miller, President and Chief Executive Officer, Abeona Therapeutics

Ken Mills, Chief Executive Officer, ReGenX Biosciences Llc

Christopher Missling, President and CEO, Anavex Life Sciences

Henry Moehring, President and CEO, Alpha-1 Foundation

Henry Moehring at World Orphan Drug Congress USA 2018

Chris Moen, President, Choroideremia Research Foundation Inc

Chris Moen at World Orphan Drug Congress USA 2018

Mehran F. Moghaddam, CEO, Orox Biosciences

Leslie Morgan, Managing Director, Durbin

Leslie Morgan at World Orphan Drug Congress USA 2018

Juliet Moritz, Executive Director, Patient Engagement & Strategic Development, Premier Research

David Mott, General Partner, N.E.A.

Tricia Mullins, Senior Director of Global Patient Advocacy, Horizon Pharma

Tricia Mullins at World Orphan Drug Congress USA 2018

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

Richard Muruve, Chief Executive Officer and President, Arch Biopartners

Joseph Musumeci, Senior Advisor, BluePrint Orphan

Joseph Musumeci at World Orphan Drug Congress USA 2018

Jack Mycka, Global President and Chief Executive Officer, Medical Marketing Economics

Jack Mycka at World Orphan Drug Congress USA 2018

Jeff Myers, CEO, Medicaid Health Plans of America

Francois Nader, Chairman, Acceleron Pharma

Yukiko Nishimura, President, NPO, Japan Patients Association, ASrid

Rodger Novak, Chief Executive Officer and Founder, CRISPR Therapeutics

Philipp Novak, Founder and Chief Executive Officer, ORPHANIX

Franz Obermayr, Chief Executive Officer, Panoptes Pharma

Tuyen Ong, Chief Development Officer, Nightstar Therapeutics

Tuyen Ong at World Orphan Drug Congress USA 2018

Ayse Deniz Ozger, Vice President, Healthcare Division, Eczacibasi Healthcare

Ayse Deniz Ozger at World Orphan Drug Congress USA 2018

Carmencita D Padilla, Founding Chairman, Philippine Society for Orphan Disorders

Samantha Parker, Chief Patient Access Officer, Lysogene

Kush Parmar, Managing Partner, 5A.M. Ventures

Douglas Paul, Vice President and Partner, Medical Marketing Economics

Douglas Paul at World Orphan Drug Congress USA 2018

Joe Payne, Founder, President & Chief Executive Officer, Arcturus Therapeutics

Mathew Pletcher, Head of Rare Disease Discovery, Roche

Mathew Pletcher at World Orphan Drug Congress USA 2018

Harsha Rajasimha, Co Founder, Organization for Rare Diseases India

Amrit Ray, Senior Vice President, Science & Medicine, Johnson & Johnson

Amrit Ray at World Orphan Drug Congress USA 2018

Luiz Fernando Lima Reis, Director of Education and Research, Hospital Sirio-Libanes

Luiz Fernando Lima Reis at World Orphan Drug Congress USA 2018

Ronald Renaud, Chief Executive Officer, Translate Bio

Ronald Renaud at World Orphan Drug Congress USA 2018

Frederic Revah, Chief Executive Officer, Genethon

Bill Rich, Past President, American Academy of Ophthalmology

Bill Rich at World Orphan Drug Congress USA 2018

Trevor Richter, Director, Common Drug Review and Optimal Use, CADTH

Betsy Ricketts, Senior Director, Government Affairs, Ultragenyx Pharmaceutical

Betsy Ricketts at World Orphan Drug Congress USA 2018

Matt Ritter, Head of Business Development, La Jolla Pharmaceutical Company

Matt Ritter at World Orphan Drug Congress USA 2018

Alan Robertson, Chief Executive Officer and Managing Director, Alsonex

Kaye Robertson, Senior Medical Officer and Medicines evaluator, Prescription Medicines Assessment Branch, Therapeutic Goods Administration

Kaye Robertson at World Orphan Drug Congress USA 2018

Chandler Robinson, CEO, Monopar Therapeutics

Stephen Rose, Chief Research Officer, Foundation Fighting Blindness

Stephen Rose at World Orphan Drug Congress USA 2018

Christer Rosén, Chairman & CEO, Jupiter Orphan Therapeutics, Inc.

Mark Rothera, President and Chief Executive Officer, Orchard Therapeutics

Tom Sabia, Vice President, Haemophilia Marketing and Operations, Sobi

Tom Sabia at World Orphan Drug Congress USA 2018

Leandro Safatle, Secretário-Executivo, ANVISA

Leandro Safatle at World Orphan Drug Congress USA 2018

Peter Saltonstall, CEO, National Organization for Rare Disorders

Jude Samulski, Vice President, Gene Therapy, Pfizer

Jude Samulski at World Orphan Drug Congress USA 2018

Gustavo San Martin, , AME - Amigos Múltiplos pela Esclerose

Lukas Scheibler, Executive Vice President of Research and Development, Acucela

Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences

Brian Schwartz, Chief Medical Officer, Arqule, Inc.

Devon Shedlock, Vice President, Preclinical Development, Poseida Therapeutics, Inc.

Devon Shedlock at World Orphan Drug Congress USA 2018

Alvin Shih, CEO, Enzyvant

Catherine Stehman Breen, Chief Medical Officer, Sarepta Therapeutics

Makoto Suematsu, Founding President, Japan Agency for Medical Research and Development

Makoto Suematsu at World Orphan Drug Congress USA 2018

Lara Sullivan, President and Founder, SpringWorks Therapeutics

Scott Sutherland, Director, Data Sciences Platform, Broad Institute of M.I.T. & Harvard

Scott Sutherland at World Orphan Drug Congress USA 2018

Ray Takigiku, President and Chief Executive Officer, Bexion Pharmaceuticals Llc

Pamela Tenaerts, Director, Clinical Trials Transformation Initiative

Vladimir Tomov, Chairman, National Alliance of People with Rare Diseases - Bulgaria

Vladimir Tomov at World Orphan Drug Congress USA 2018

Roberto Tonelli, President, Chairman of the Board, Co-founder, BIOGENERA

Arthur Tzianabos, President and Chief Executive Officer, Homology Medicines

Tim Van Hauwermeiren, Chief Executive Officer, Argenx

Tim Van Hauwermeiren at World Orphan Drug Congress USA 2018

Yvette Venable, International Public Affairs Lead, Kyowa Kirin International

Yvette Venable at World Orphan Drug Congress USA 2018

Bryan Viau, Chairperson, The Huntington’s Disease Youth Organization

Bryan Viau at World Orphan Drug Congress USA 2018

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

Sue Washer, President/CEO, AGTC

Christina Waters, CEO and Founder, RARE Science

Christina Waters at World Orphan Drug Congress USA 2018

Martin Welschof, Chief Executive Officer, Opsona Therapeutics Ltd

Durhane Wong-Rieger, President and Chief Executive Officer, Canadian Organization For Rare Disorders

Stephen Wright, Chief Medical Officer, GW Pharmaceuticals

Stephen Wright at World Orphan Drug Congress USA 2018

Barbara Wuebbels, Vice President of Patient Advocacy, Audentes Therapeutics

Nora Yang, Portfolio and Project Management, Strategic Operations, National Institutes of Health

 

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