2018 Speakers

 

Karen Aiach, Chief Executive Officer, Lysogene

Tero-Pekka Alastalo, President, Chief Medical Officer, Blueprint Genetics

Tero-Pekka Alastalo at World Orphan Drug Congress USA 2018

Charlie Albright, Chief Scientific Officer, Editas Medicine

Charlie Albright at World Orphan Drug Congress USA 2018

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

Daniel Anderson, Head of Commercial Partnerships, Invitae

Daniel Anderson at World Orphan Drug Congress USA 2018

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

Francis Arickx, Head of the Directorate Reimbursement of Medicines and Pharmaceutical Policy,, National Institute for Health and Disability Insurance RIZIV-INAMI

Francis Arickx at World Orphan Drug Congress USA 2018

Heather Ascani, Business Operations, Applied Systems Biology Core Internal Medicine – Nephrology, University of Michigan

Heather Ascani at World Orphan Drug Congress USA 2018

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

Christopher Austin, Director of National Center and Advancing Translational Sciences, National Institutes of Health

Christopher Austin at World Orphan Drug Congress USA 2018

Mark Baglin, Vice President of Global Marketing, Alnylam Pharmaceuticals

Mark Baglin at World Orphan Drug Congress USA 2018

Mariah Baltezegar, Executive Director Clinical Development, Syneos Health

Mariah Baltezegar at World Orphan Drug Congress USA 2018

Jarbas Barbosa da Silva Jr, Director-President, ANVISA

Jarbas Barbosa da Silva Jr 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, Professor, New York University Langone Medical Center

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

Silke Baumann, Head of Division, Medicinal Product Supply in the Statutory Health Insurance, Federal Ministry of Health Germany

Silke Baumann at World Orphan Drug Congress USA 2018

Denis Belyakov, Executive Director, 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

Stella Blackburn, Vice President, Global Head of Early Access and Risk Management, IQVIA

Stella Blackburn 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

Aldar Bourinbaiar, Chief Executive Officer, Immunitor Inc

Steven Brenner, Professor for Plant and Microbial Biology, University Of California, Berkeley

Steven Brenner at World Orphan Drug Congress USA 2018

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, Director of Outreach for the Rare and Undiagnosed Network, The Atypical HUS Foundation

Anne Bruns at World Orphan Drug Congress USA 2018

Raquel Cabo, VP, Global Mkt Access, Ovid Therapeutics

Raquel Cabo at World Orphan Drug Congress USA 2018

Jean Campbell, Principal, J.F. Campbell Consultants Llc

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

David Carey, Associate Chief Research Officer, Geisinger Health System

David Carey at World Orphan Drug Congress USA 2018

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

Christine Charman, External Asset Lead, Takeda Development Centre Europe

Christine Charman at World Orphan Drug Congress USA 2018

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

Walter Colasante, C.R.A. Charles River Associates

Walter Colasante at World Orphan Drug Congress USA 2018

Matt Comstock, Vice President Of US Business Development, WEP Clinical

Matt Comstock at World Orphan Drug Congress USA 2018

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, FDA

David Cory, President, Chief Executive Officer, Eiger Biopharmaceuticals

Don Creighton, Divisional Principal, Icon

Don Creighton at World Orphan Drug Congress USA 2018

Anne Cropp, Chief Scientific Officer, Early Access Care

Anne Cropp at World Orphan Drug Congress USA 2018

Gregory Daniel, Deputy Director, Duke University

Olivier Danos, Chief Scientific Officer, Regenxbio Inc

Mark Dant, Executive Director, The Ryan Foundation

Mark Dant at World Orphan Drug Congress USA 2018

Daniel De Boer, Chief Executive Officer, Proqr

Bob Desai, Vice President, Technical, Parexel International Corp

Robert Donnell, Director of Business Development, Durbin

Robert Donnell at World Orphan Drug Congress USA 2018

Dan Donovan, Founder and Chief Executive Officer, rareLife solutions

Dan Donovan at World Orphan Drug Congress USA 2018

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

Cindy Dukes, VP, Global Project Management, Vaccines, Rare Disease, ICON

Cindy Dukes at World Orphan Drug Congress USA 2018

Mark Dunning, Chairman, Coalition for Usher Syndrome Research

Mark Dunning at World Orphan Drug Congress USA 2018

Kelly East, Genetic Counselor, Hudson Alpha Institute for Biotechnology

Kelly East at World Orphan Drug Congress USA 2018

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

Sam Emaminejad, Assistant Professor, Electrical Engineering, UCLA

Sam Emaminejad at World Orphan Drug Congress USA 2018

Offer Fabian, Chief Executive Officer and Co Founder, Musli Thyropeutics

Offer Fabian at World Orphan Drug Congress USA 2018

Mariana Fagnilli, Head of Global Patient Advocacy, bluebird bio

Mariana Fagnilli at World Orphan Drug Congress USA 2018

Erin Farris, Director, Project Management, Atlantic Research Group Inc

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

Kelly Franchetti, Vice President, Global Patients Insights and Engagement, Mapi Group

Kelly Franchetti at World Orphan Drug Congress USA 2018

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

Gary Geipel, Senior Director, Global Brand Communications, Ultragenyx Pharmaceutical

Gary Geipel at World Orphan Drug Congress USA 2018

Jayne Gershkowitz, Chief Patient Advocate, Amicus Therapeutics

Martin Giedlin, Vice President, Technical Operations, Poseida Therapeutics, Inc.

Martin Giedlin at World Orphan Drug Congress USA 2018

Gretchen Goller, VP, Patient Recruitment, ICON

Gretchen Goller at World Orphan Drug Congress USA 2018

Thomas Goss, Senior Vice President, Boston Healthcare Associates Inc

Anil Goud Jegga, Assistant Professor, Cincinnati Children's Hospital Medical Center

Anil Goud Jegga at World Orphan Drug Congress USA 2018

Rory Graham, Regulatory, Mapi Group

Rory Graham at World Orphan Drug Congress USA 2018

Scott Gray, Chief Executive Officer, Clincierge

Peter Greenleaf, Chief Executive Officer, Sucampo Pharmaceuticals Inc

Carol Greve-Phillips, Strategic Advisory Board Member, Atlantic Research Group Inc

Philippe Guedat, Managing Director, In Flectis Bio Science

Tim Guilliams, CEO, Healx

Tim Guilliams at World Orphan Drug Congress USA 2018

Rodolphe Hajj, Chief Pharmacology Officer, Pharnext

Rodolphe Hajj at World Orphan Drug Congress USA 2018

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

Beverly Harrison, Head, Patient Support, Janssen Office of the Chief Medical Officer, Janssen Research and Development Llc

Beverly Harrison at World Orphan Drug Congress USA 2018

Mark Herbert, Interim President, Arcturus Therapeutics

Mark Herbert at World Orphan Drug Congress USA 2018

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

Kenneth Hobby, President, Cure SMA

Melissa Hogan, Founder and President, Project Alive

Melissa Hogan at World Orphan Drug Congress USA 2018

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

Tony Howell, Co-Founder & COO, rareLife solutions

Tony Howell 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 Partner, Atheneos Ventures and CEO, Auxesia Orion

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

Kevin Kissling, Vice President, 3PL Services, McKesson Specialty Health

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

Matthias Kretzler, Professor of Internal Medicine, Computational Medicine and Bioinformatics, University of Michigan

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

Kathryn Lasch, Executive Director and Patient Reported Outcomes, Pharmerit International

Kathryn Lasch at World Orphan Drug Congress USA 2018

Yann Le Cam, Chief Executive Officer, EURORDIS

Nadea Leavitt, Predictive Analytics Principal, Real-World Insights, IQVIA

Nadea Leavitt at World Orphan Drug Congress USA 2018

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

Dana Levasseur, Leader and Associate Director, Bioverativ

Dana Levasseur at World Orphan Drug Congress USA 2018

Eric Li, Chief Scientific Officer, Luxena Pharmaceuticals

Jan Liliemark, Professor, Program Manager, Swedish Council on Health Technology Assessment

Sook Yee Lim, Vice President, Malaysian Rare Disorders Society

Sook Yee 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

Sandy Macrae, President and Chief Executive Officer, Sangamo Therapeutics

Jay Madan, President, Innovate Biopharmaceuticals Inc

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

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

Jordanna Mora, Associate Director of Patient Advocacy and Engagement, Alnylam Pharmaceuticals

Jordanna Mora at World Orphan Drug Congress USA 2018

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

Tricia Mullins, Board Member, Neuromuscular Disease Foundation

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

Michelle Muscat, Co-Founder and President, National Alliance for Rare Diseases Support - Malta

Michelle Muscat at World Orphan Drug Congress USA 2018

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

Marilee Newman, Director, Data Management, Atlantic Research Group Inc

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

Rodger Novak, Chief Executive Officer and Founder, CRISPR Therapeutics

Franz Obermayr, Chief Executive Officer, Panoptes Pharma

Hugh O'Neill, President, Autoimmune & Rare Disease, Mallinckrodt Pharmaceuticals

Hugh O'Neill at World Orphan Drug Congress USA 2018

Tuyen Ong, Chief Development Officer, Nightstar Therapeutics

Tuyen Ong at World Orphan Drug Congress USA 2018

Colin Orford, EVP Drug Development, ICON

Colin Orford 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

Catherine Pajak, Senior Director, Marketing, North America, Lysosomal Storage Disorders, BioMarin Pharmaceutical Inc.

Catherine Pajak at World Orphan Drug Congress USA 2018

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

Tanya Potashnik, Director Policy & Economic Analysis, Patented Medicine Prices Review Board, Goverment Of Canada

Tanya Potashnik at World Orphan Drug Congress USA 2018

Chad Pulliam, Director, Channel Operations,, Jazz Pharmaceuticals

Harsha Rajasimha, Co Founder, Organization for Rare Diseases India

Julie Raskin, Executive Director, Congenital Hyperinsulinism International

Julie Raskin 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

Erik Richardson, Project Leader of Gene Therapy, Acucela

Erik Richardson 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

John Ripple, Chief Executive Officer, Exonics

John Ripple 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

Rhonda Roberts, Associate Director, Biostatistics, Atlantic Research Group Inc

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

Angi Robinson, Executive Director, Strategic Development, Rare Disease & Pediatrics, Premier Research

Angi Robinson at World Orphan Drug Congress USA 2018

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

Mark Rothera, President and Chief Executive Officer, Orchard Therapeutics

Stephanie Roy, Principal, Real-World Evidence Solutions, IQVIA

Stephanie Roy 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

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

Brian Schwartz, Chief Medical Officer, Arqule, Inc.

Simone Seiter, Vice President, IQVIA

Simone Seiter at World Orphan Drug Congress USA 2018

Alvin Shih, CEO, Enzyvant

Jaswinder Singh Khera, Managing Director, WEP Clinical

Jaswinder Singh Khera at World Orphan Drug Congress USA 2018

Alexander Stephan, Principal Scientist, Rare Disease - Discovery, Roche

Alexander Stephan at World Orphan Drug Congress USA 2018

Makoto Suematsu, Founding President, AMED

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

Cynthia Tifft, Deputy Clinical Director, National Human Genome Research Institute

Cynthia Tifft at World Orphan Drug Congress USA 2018

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

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

Bob Ward, Chairman of the Board and Chief Executive Officer, Eloxx Pharmaceuticals

Bob Ward at World Orphan Drug Congress USA 2018

Sue Washer, President/CEO, AGTC

Martin Welschof, Chief Executive Officer, Opsona Therapeutics Ltd

Brad Whitlow, Senior Project Manager, Atlantic Research Group Inc

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

Leung-Ming Yu, Scientific officer HTA and reimbursement, Norwegian Medicines Agency

Leung-Ming Yu at World Orphan Drug Congress USA 2018

Martine Zimmermann, SVP, Head of Global Regulatory Affairs, Alexion Pharmaceuticals

Martine Zimmermann at World Orphan Drug Congress USA 2018

 

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