2018 speakers

 

Karen Aiach, Chief Executive Officer, Lysogene

Karen Aiach at World Orphan Drug Congress USA 2018

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

Ronald Alfa, Director of Translational Biology, Recursion Pharmaceuticals

Ronald Alfa at World Orphan Drug Congress USA 2018

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, R&D Rare Diseases Unit Head, 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)

Katri Asikainen at World Orphan Drug Congress USA 2018

Leone Atkinson, Executive Medical Director, Covance

Leone Atkinson at World Orphan Drug Congress USA 2018

Christopher Austin, Director, National Center for Advancing Translational Sciences (NCATS), National Center for Advancing Translational Sciences - NCATS

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

Jeanne Barnett at World Orphan Drug Congress USA 2018

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

Ron Bartek at World Orphan Drug Congress USA 2018

Alison Bateman House, Professor, New York University Langone Medical Center

Alison Bateman House at World Orphan Drug Congress USA 2018

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

Saira Bates at World Orphan Drug Congress USA 2018

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

Denis Belyakov at World Orphan Drug Congress USA 2018

Carole Ben Maimon, Chief Executive Officer, Chondrial Therapeutics

Carole Ben Maimon at World Orphan Drug Congress USA 2018

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

Laxminarayan Bhat at World Orphan Drug Congress USA 2018

Sonal Bhatia, Vice President, North America Medical Lead, Pfizer Rare Disease

Sonal Bhatia at World Orphan Drug Congress USA 2018

Dawn Bir, Chief Commercial Officer, Reata Pharmaceuticals

Dawn Bir at World Orphan Drug Congress USA 2018

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-Orphan Drug Unit

Stella Blackburn at World Orphan Drug Congress USA 2018

Bruce Bloom, President, Cures Within Reach

Bruce Bloom at World Orphan Drug Congress USA 2018

Betsy Bogard, Senior Director of Medical Affairs, bluebird bio

Betsy Bogard at World Orphan Drug Congress USA 2018

David Bonita, Private Equity Partner, Orbimed Advisors

David Bonita at World Orphan Drug Congress USA 2018

Aldar Bourinbaiar, Chief Executive Officer, Immunitor Inc

Aldar Bourinbaiar at World Orphan Drug Congress USA 2018

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

Ari Brettman at World Orphan Drug Congress USA 2018

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

Brian Bronk at World Orphan Drug Congress USA 2018

Philip J. Brooks, program director, National Center for Advancing Translational Sciences - NCATS

Philip J. Brooks at World Orphan Drug Congress USA 2018

Bert Bruce, Vice President, Rare Disease Commercial Development, Pfizer

Bert Bruce at World Orphan Drug Congress USA 2018

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

Anne Bruns at World Orphan Drug Congress USA 2018

Shannon Burkoth, Advocate in the Spotlight, Rare Advocacy Movement

Shannon Burkoth at World Orphan Drug Congress USA 2018

Jim Burns, President and Chief Executive Officer, Casebia Therapeutics

Jim Burns at World Orphan Drug Congress USA 2018

Raquel Cabo, Vice President, Global Market Access, Ovid Therapeutics

Raquel Cabo at World Orphan Drug Congress USA 2018

Jean Campbell, Strategic Advisory Board Member, Atlantic Research Group Inc

Jean Campbell at World Orphan Drug Congress USA 2018

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

Preston Campbell at World Orphan Drug Congress USA 2018

David Carey, Associate Chief Research Officer, Geisinger Health System

David Carey at World Orphan Drug Congress USA 2018

Mary Ellen Carroll, Director, Global Commercial Development, Rare Diseases, Pfizer

Mary Ellen Carroll at World Orphan Drug Congress USA 2018

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

Rick Chapman at World Orphan Drug Congress USA 2018

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, Vice President, 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, Senior 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

Jacqueline Corrigan-Curay at World Orphan Drug Congress USA 2018

David Cory, President, Chief Executive Officer, Eiger Biopharmaceuticals

David Cory 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

Gregory Daniel at World Orphan Drug Congress USA 2018

Olivier Danos, Chief Scientific Officer, Regenxbio Inc

Olivier Danos at World Orphan Drug Congress USA 2018

Mark Dant, Executive Director, The Ryan Foundation

Mark Dant at World Orphan Drug Congress USA 2018

Bruce Daugherty, Chief Scientific Officer, Targazyme

Bruce Daugherty at World Orphan Drug Congress USA 2018

Daniel De Boer, Chief Executive Officer, Proqr

Daniel De Boer at World Orphan Drug Congress USA 2018

Bob Desai, Vice President, Technical, PAREXEL International Corp

Bob Desai at World Orphan Drug Congress USA 2018

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

Ashish Dugar, Vice President, Global Medical Affairs, Homology Medicines

Ashish Dugar at World Orphan Drug Congress USA 2018

Cynthia Dukes, Vice President - Global Project Management, ICON plc

Cynthia Dukes at World Orphan Drug Congress USA 2018

Mark Dunning, Chairman, Usher Syndrome Coalition

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

Emeka Egwim, Pharmacist, Center for Medicare & Medicaid Services - CMS

Emeka Egwim at World Orphan Drug Congress USA 2018

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

David Elvira Martinez at World Orphan Drug Congress USA 2018

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

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

Erin Farris at World Orphan Drug Congress USA 2018

Beata Ferencz, Project Manager, Rare Diseases Sweden

Beata Ferencz at World Orphan Drug Congress USA 2018

Kelly Franchetti, Vice President, Global Patients Insights and Engagement, Mapi, an ICON plc company

Kelly Franchetti at World Orphan Drug Congress USA 2018

Stephen Franklin, Chief Executive, Evgen Pharma

Stephen Franklin at World Orphan Drug Congress USA 2018

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

Pat Furlong at World Orphan Drug Congress USA 2018

Prasad Gabbita, Chief Executive Officer, P2d Inc

Prasad Gabbita at World Orphan Drug Congress USA 2018

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

Pamela Gavin at World Orphan Drug Congress USA 2018

Mary Jane Geiger, VP, Drug Development, Cardiology, ICON plc

Mary Jane Geiger at World Orphan Drug Congress USA 2018

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

Jayne Gershkowitz at World Orphan Drug Congress USA 2018

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

Martin Giedlin at World Orphan Drug Congress USA 2018

Abraham Gitterman, Attorney, Arnold & Porter

Abraham Gitterman at World Orphan Drug Congress USA 2018

Gretchen Goller, VP, Patient Recruitment, ICON plc

Gretchen Goller at World Orphan Drug Congress USA 2018

Thomas Goss, Senior Vice President, Boston Healthcare Associates Inc

Thomas Goss at World Orphan Drug Congress USA 2018

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

Anil Goud Jegga at World Orphan Drug Congress USA 2018

Rory Graham, Senior Director, EU Regulatory Services, Mapi, an ICON plc company

Rory Graham at World Orphan Drug Congress USA 2018

Scott Gray, Chief Executive Officer, Clincierge

Scott Gray at World Orphan Drug Congress USA 2018

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

Carol Greve-Phillips at World Orphan Drug Congress USA 2018

Philippe Guedat, Chief Executive Officer, InFlectis BioScience

Philippe Guedat at World Orphan Drug Congress USA 2018

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

Rylan Hanks at World Orphan Drug Congress USA 2018

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

Michael Heartlein, Chief Technical Officer, Translate Bio

Michael Heartlein at World Orphan Drug Congress USA 2018

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

Katherine High at World Orphan Drug Congress USA 2018

Kenneth Hobby, President, Cure SMA

Kenneth Hobby at World Orphan Drug Congress USA 2018

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

Birthe Byskov Holm at World Orphan Drug Congress USA 2018

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

TaLana Hughes, Executive Director, Sickle Cell Disease Association of Illinois

TaLana Hughes at World Orphan Drug Congress USA 2018

Adam Hutchings, Managing Director, Dolon

Adam Hutchings at World Orphan Drug Congress USA 2018

Douglas Ingram, Chief Executive Officer, Sarepta Therapeutics

Douglas Ingram at World Orphan Drug Congress USA 2018

Fred Jacobs, Chief Executive Officer and Co Founder, TYG

Fred Jacobs at World Orphan Drug Congress USA 2018

Julia Jenkins, Executive Director, Everylife Foundation

Julia Jenkins at World Orphan Drug Congress USA 2018

Edmund Jessop, Medical Adviser, National Health Service

Edmund Jessop at World Orphan Drug Congress USA 2018

Steven Kaminsky, Chief Science Officer, International Rett Syndrome Foundation

Steven Kaminsky at World Orphan Drug Congress USA 2018

Vikram Karnani, Chief Commercial Ofiicer, Horizon Pharma

Vikram Karnani at World Orphan Drug Congress USA 2018

Karen Kaucic, Senior Vice President, Global Head of Early Development, PPD

Karen Kaucic at World Orphan Drug Congress USA 2018

Kazem Kazempour, President and Chief Executive Officer, Amarex Clinical Research

Kazem Kazempour at World Orphan Drug Congress USA 2018

M Ken Kengatharan, Managing Partner, Atheneos Ventures and CEO, Auxesia Orion

M Ken Kengatharan at World Orphan Drug Congress USA 2018

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

Alastair Kent OBE at World Orphan Drug Congress USA 2018

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

Kevin Kissling at World Orphan Drug Congress USA 2018

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

Cristina Klafehn at World Orphan Drug Congress USA 2018

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

Terri Klein at World Orphan Drug Congress USA 2018

Lawrence Klein, Head of Business Development and Strategy, CRISPR Therapeutics

Lawrence Klein at World Orphan Drug Congress USA 2018

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

Diane Kleinermans at World Orphan Drug Congress USA 2018

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

Matthias Kretzler at World Orphan Drug Congress USA 2018

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

Alain Lamproye, Chief Executive Officer, YposKesi

Alain Lamproye at World Orphan Drug Congress USA 2018

David Lapidus, President, Lapidus Data

David Lapidus at World Orphan Drug Congress USA 2018

Kathryn Lasch, Executive Director of Patient-Reported Outcomes, Pharmerit International

Kathryn Lasch at World Orphan Drug Congress USA 2018

Yann Le Cam, Chief Executive Officer, EURORDIS

Yann Le Cam at World Orphan Drug Congress USA 2018

Leslie Leahy, Vice President, Medical Sciences, Ovid Therapeutics

Leslie Leahy at World Orphan Drug Congress USA 2018

Nadea Leavitt, Predictive Analytics Principal, Real-World Insights, IQVIA-Orphan Drug Unit

Nadea Leavitt at World Orphan Drug Congress USA 2018

Dana Levasseur, Leader and Associate Director, Bioverativ

Dana Levasseur at World Orphan Drug Congress USA 2018

Debra Lewis, Acting Director, Office of Orphan Products, U.S. Food and Drug Administration

Debra Lewis at World Orphan Drug Congress USA 2018

Eric Li, Chief Scientific Officer, Luxena Pharmaceuticals

Eric Li at World Orphan Drug Congress USA 2018

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

Jan Liliemark at World Orphan Drug Congress USA 2018

Sook Yee Lim, Co-founder, We CARE Journey

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.

Igor Lokot at World Orphan Drug Congress USA 2018

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

Aoife M Brennan at World Orphan Drug Congress USA 2018

Sandy Macrae, President and Chief Executive Officer, Sangamo Therapeutics

Sandy Macrae at World Orphan Drug Congress USA 2018

Jay Madan, President, Innovate Biopharmaceuticals Inc

Jay Madan at World Orphan Drug Congress USA 2018

Will Maier, Chief Scientific Officer, ICON plc

Will Maier at World Orphan Drug Congress USA 2018

Helen Malherbe, Board Member, Genetic Alliance South Africa

Helen Malherbe at World Orphan Drug Congress USA 2018

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

Catherine Martin at World Orphan Drug Congress USA 2018

John Maslowski, CEO, Fibrocell Science Inc

John Maslowski at World Orphan Drug Congress USA 2018

Holly May, Vice President, Sales and Marketing, Sobi

Holly May at World Orphan Drug Congress USA 2018

Francesca Mcbride, Director Regulatory Compliance, Jacobs

Francesca Mcbride at World Orphan Drug Congress USA 2018

Cameron McDaniel, Scientific Contributor, Arch Biopartners

Cameron McDaniel 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

Donnie McGrath at World Orphan Drug Congress USA 2018

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

Ken Mills, Chief Executive Officer, ReGenX Biosciences Llc

Ken Mills at World Orphan Drug Congress USA 2018

Christopher Missling, President and CEO, Anavex Life Sciences

Christopher Missling at World Orphan Drug Congress USA 2018

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

Mehran F. Moghaddam at World Orphan Drug Congress USA 2018

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

Jordanna Mora at World Orphan Drug Congress USA 2018

Trista Morrison, Vice President of Communications, Sobi

Trista Morrison at World Orphan Drug Congress USA 2018

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

David Mott at World Orphan Drug Congress USA 2018

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

Michael Murphy at World Orphan Drug Congress USA 2018

Samantha Murphy, Associate Director of Business Development, Arcturus Therapeutics

Samantha Murphy 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

Jeff Myers at World Orphan Drug Congress USA 2018

Francois Nader, Chairman, Acceleron Pharma

Francois Nader at World Orphan Drug Congress USA 2018

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

Marilee Newman at World Orphan Drug Congress USA 2018

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

Yukiko Nishimura at World Orphan Drug Congress USA 2018

Franz Obermayr, Chief Executive Officer, Panoptes Pharma

Franz Obermayr at World Orphan Drug Congress USA 2018

Steve Orndorff, Chief Executive Officer and Co Founder, IM Therapeutics

Steve Orndorff at World Orphan Drug Congress USA 2018

Ayse Deniz Ozger, In-House Senior Business Development Consultant, Eczacibasi Investment & Healthcare Co.

Ayse Deniz Ozger at World Orphan Drug Congress USA 2018

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

Carmencita D Padilla at World Orphan Drug Congress USA 2018

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

Samantha Parker at World Orphan Drug Congress USA 2018

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

Kush Parmar at World Orphan Drug Congress USA 2018

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

Sarah Procario, Advocacy-Communications Manager, The Hemophilia Foundation of Michigan

Sarah Procario at World Orphan Drug Congress USA 2018

Chad Pulliam, Director, Channel Operations, Jazz Pharmaceuticals

Chad Pulliam at World Orphan Drug Congress USA 2018

Harsha Rajasimha, Co Founder, Organization for Rare Diseases India

Harsha Rajasimha at World Orphan Drug Congress USA 2018

Julie Raskin, Executive Director, Congenital Hyperinsulinism International

Julie Raskin at World Orphan Drug Congress USA 2018

Frederic Revah, Chief Executive Officer, Genethon

Frederic Revah at World Orphan Drug Congress USA 2018

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

Trevor Richter at World Orphan Drug Congress USA 2018

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

Rhonda Roberts at World Orphan Drug Congress USA 2018

Kaye Robertson, Assistant Director Prescription Medicines Clinical Evaluation Unit 3, Therapeutic Goods Administration

Kaye Robertson at World Orphan Drug Congress USA 2018

Alan Robertson, Chief Executive Officer and Managing Director, Alsonex

Alan Robertson at World Orphan Drug Congress USA 2018

Chandler Robinson, CEO, Monopar Therapeutics

Chandler Robinson at World Orphan Drug Congress USA 2018

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

Angi Robinson at World Orphan Drug Congress USA 2018

Gregory Robinson, Chief Scientific Officer, Nightstar Therapeutics

Gregory Robinson at World Orphan Drug Congress USA 2018

Luke Rosen, Associate Director, Patient Engagement, Ovid Therapeutics

Luke Rosen at World Orphan Drug Congress USA 2018

Christer Rosen, CEO, Jupiter Orphan Therapeutics, Inc.

Christer Rosen at World Orphan Drug Congress USA 2018

Mark Rothera, President and Chief Executive Officer, Orchard Therapeutics

Mark Rothera at World Orphan Drug Congress USA 2018

Stephanie Roy, Principal, Real-World Evidence Solutions, IQVIA-Orphan Drug Unit

Stephanie Roy at World Orphan Drug Congress USA 2018

Rebecca Rupert, Vice President, Global Marketing, Argenx

Rebecca Rupert 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

Peter Saltonstall at World Orphan Drug Congress USA 2018

Gustavo San Martin, AME - Amigos Múltiplos pela Esclerose

Gustavo San Martin at World Orphan Drug Congress USA 2018

Josh Schafer, General Manager, International and VP,Business Development & Licensing, Mallinckrodt Pharmaceuticals

Josh Schafer at World Orphan Drug Congress USA 2018

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

Scott Schliebner at World Orphan Drug Congress USA 2018

Brian Schwartz, Chief Medical Officer, Arqule, Inc.

Brian Schwartz at World Orphan Drug Congress USA 2018

Simone Seiter, Vice President Global Lead Launch Excellence, IQVIA-Orphan Drug Unit

Simone Seiter at World Orphan Drug Congress USA 2018

Neal Sharpe, VP Translational Sciences, Eloxx Pharmaceuticals

Neal Sharpe at World Orphan Drug Congress USA 2018

Alvin Shih, CEO, Enzyvant

Alvin Shih at World Orphan Drug Congress USA 2018

Jaswinder Singh Khera, Managing Director, WEP Clinical

Jaswinder Singh Khera at World Orphan Drug Congress USA 2018

Krupa Sivamurthy, Medical Director, Pfizer

Krupa Sivamurthy at World Orphan Drug Congress USA 2018

Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer

Bob Smith at World Orphan Drug Congress USA 2018

David Solomon, Chief Executive Officer, Akari TX

David Solomon at World Orphan Drug Congress USA 2018

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

Alexander Stephan at World Orphan Drug Congress USA 2018

Lara Sullivan, President and Founder, SpringWorks Therapeutics

Lara Sullivan at World Orphan Drug Congress USA 2018

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

Ray Takigiku at World Orphan Drug Congress USA 2018

Pamela Tenaerts, Executive Director, Clinical Trials Transformation Initiative

Pamela Tenaerts at World Orphan Drug Congress USA 2018

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

Roberto Tonelli at World Orphan Drug Congress USA 2018

Arthur Tzianabos, President and Chief Executive Officer, Homology Medicines

Arthur Tzianabos at World Orphan Drug Congress USA 2018

Tiina Urv, Program Director, Office of Rare Disease Research, National Center for Translational Sciences, National Institute of Health

Tiina Urv at World Orphan Drug Congress USA 2018

James Valentine, Attorney, Hyman Phelps and Mcnamara P.C.

James Valentine 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

Sue Washer, President/CEO, AGTC

Sue Washer at World Orphan Drug Congress USA 2018

Christina Waters, Senior Vice President and General Manager, Rare Disease Programs, WuXi NextCODE

Christina Waters at World Orphan Drug Congress USA 2018

Martin Welschof, Chief Executive Officer, Opsona Therapeutics Ltd

Martin Welschof at World Orphan Drug Congress USA 2018

Brad Whitlow, Senior Project Manager, Atlantic Research Group Inc

Brad Whitlow at World Orphan Drug Congress USA 2018

Durhane Wong-Rieger, President, Canadian Organization For Rare Disorders

Durhane Wong-Rieger at World Orphan Drug Congress USA 2018

Howard Woolley, President, Howard Woolley Group LLC

Howard Woolley at World Orphan Drug Congress USA 2018

Barbara Wuebbels, Vice President of Patient Advocacy, Audentes Therapeutics

Barbara Wuebbels at World Orphan Drug Congress USA 2018

Laura Wuerth, EVP and Rare Strategies Leader, rareLife solutions

Laura Wuerth at World Orphan Drug Congress USA 2018

Nora Yang, Director, Portfolio and Project Management, Strategic Operations, Division of Pre-Clinical Innovation, National Center for Advancing Translational Sciences - NCATS

Nora Yang at World Orphan Drug Congress USA 2018

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