2023 Speakers
Faruk Abdullah, President, Professional Services and Chief Business Officer, EVERSANA
Suchismita Acharya, Chief Executive Officer, AyuVis Research
Rania Al Shami, Chief Business and Strategy Officer, PDC CRO
Meg Alexander, Corporate Affairs Officer, Ovid Therapeutics, Ovid Therapeutics
Paul Aliu, Head Global Governance Office, Novartis Chief Medical Office (CMO), Novartis
Simon Andrews, Senior Vice President, Head of Analytics, EVERSANA
Irina Antonijevic, Chief Medical Officer, EveryONE Medicines
Andrew Auruku, Chief Executive Officer, Rare Diseases Uganda-RDU
Rena Baek, Senior Director - Rare Diseases, Moderna, Inc.
Ritu Baral, Managing Director And Senior Biotechnology Analyst, Cowen
Kelly Barta, President, Coalition of Skin Diseases
Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance
Norman Barton, Chair of the Clinical and Scientific Advisory Board, Oak Hill Bio
Kevin Baruzzi, Partner, Simon-Kucher
Mark Battaglini, Chief Strategy Officer, Alliance for Regenerative Medicine
Wendy Benson, Chief Strategy and Innovation Officer, Rady Children's Institute for Genomic Medicine
Scott Bergeron, Partner, Clinical Research, iTakeControl, a Red Nucleus Company
Dawn Berry, President and Co-Founder, LunaPBC
Sulagna Bhattacharya, Chief Executive Officer, NanoScope Technologies
Kathie Bishop, SVP, Head of Rare Disease and External Innovation, Acadia Pharmaceuticals Inc.
Henric Bjarke, Chief Operating Officer, Inozyme
Fabrice Bocquillon, Global Head, Digital Specialty Care, Sanofi
Natasha Bonhomme, Chief Strategy Officer, Genetic Alliance Founder, Expecting Health
Wendy Borsari, Senior Manager of Patient Advocacy, Tenaya Therapeutics
Pierre-Olivier Boyer, Country Manager, PTC Therapeutics
Marie Brace, Patient Access Liaison, Catalyst Pharmaceutical Partners
Julie Breneiser, Executive Director, Gorlin Syndrome Alliance
Matthias Breugelmans, Chief Executive Officer, Elastrin Therapeutics
Rusty Bromley, Director of Operations, AyuVis Research, Inc.
P.J. Brooks, Acting Director, Division of Rare Diseases Research Innovation, NIH/NCATS
Robert Buchanan, Founder And Partner, Ventac Partners
Raquel Cabo, VP, Global Head of Pricing & Market Access, Ovid Therapeutics
Robert M. Califf, Commissioner, U.S. Food and Drug Administration
Leonardo Calzetti, Co-founding Director & Sr. Partner, BlueDil International
Tanisha Carino, Board Member, Every Cure
Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics
John Cavitt, Parent, BCM Families Foundation
Afshawn Chakamian, Director of Therapeutic Expertise, ICON
Anne-Sophie Chalandon, Head of Rare Diseases Global Public Affairs Rare Disease and ATMP Policy at Sanofi, Vice-Chair of the EFPIA OMP WG, Sanofi
David Chapman, Vice President of Market Access, Agios Pharmaceuticals
Richard Chapman, Chief Science Officer, Innovation and Value Initiative
Jonathan CHAPPER, CHAPPER healthcare - CEO, EUROPACCESS
Yi-Chun Maria Chen, VP of Business Development, BRIM Biotechnology
Nimi Chhina, Head, Global R&D and Regulatory Policy, BioMarin Pharmaceutical Inc.
Nitin Choudhary, Vice President, IPM.ai
Steven Cohen, Executive Director, Translational Medicine-Oncology & Internal Medicine, & Search & Evaluation-IM, Daiichi Sankyo
Cathy Collet, ALS Caregiver, Patient Advocate
Rachelle Cook, Community Manager, TREND Community
Ashley Cox, Senior Principal, Head of Europe | President HBA - London Chapter, EVERSANA
Laura Cox, Director Partnerships, myTomorrows
Carole Cramer, Chief Executive Officer and CoFounder, Biostrategies
Joslyn Crowe, Executive Director, National Niemann Pick Disease Foundation Inc
Charlotte Damiano, Global Regulatory Partner, Life Sciences, Hogan Lovells
Mark Dant, Executive Director, The Ryan Foundation
Kristina Davidson, Chief Corporate Affairs Officer, Horizon Therapeutics
Kendall Davis, Director, Advocacy & Engagement Strategy Center for Rare Diseases, ICON
Michelle Davis, Executive Director, International Fibrodysplasia Ossificans Progressiva Association
Sugato De, VP, Regulatory Strategy, Head of MedTech, Allucent
Laurence Dean, Managing Member, Twin Spires Consulting LLC
Tom Defay, Deputy Head Diagnostics Strategy & Development, Alexion Pharmaceuticals
Christopher DeFelice, Founder and CTO, TREND Community
Maria Della Rocca, Sr. Director, Patient Support & Education, Global Genes
Lisa Deschamps, Chief Executive Officer, AviadoBio
Patricia Dickson, Centennial Professor of Pediatrics, Professor of Genetics, Chief, Division of Genetics and Genomic Medicine, Washington University
Deb Discenza, Chief Executive Officer, PreemieWorld
Victoria Dohnal, Associate Director, Global Regulatory Policy & Intelligence, Janssen
Chowdary Dondapati, Executive Director, US Head of Diagnostic Marketing, Precision Medicine, BAYER
Danielle Dong, Global Operations and Advocacy Lead, Rare Disease Registries, Sanofi
Kate Donigan, Senior Director, Science & Regulatory Policy, Global Policy, Government, & Patient Affairs, Sarepta Therapeutics
Michael Dudley, Chief Executive Officer and Co-founder, TransCode Therapeutics
Stephanie Duffy, Global Patient Advocacy and Engagement Director, The Janssen Pharmaceutical Company of Johnson and Johnson
Patrick Dunn, Executive Director, International Pemphigus Pemphigoid Foundation
Abie Ekangaki, Vice President, Statistical Consulting, Premier Research
Sean Ekins, Chief Executive Officer, Collaborations Pharmaceuticals
Shaun Ellis, Vice President, Global Market Access CVM Retina, Janssen (J&J)
Ben Enejo, Partner - Life Sciences, Arthur D. Little
Wendy Erler, Vice President, Global Head Patient Experience & Insights, Alexion – AstraZeneca Rare Disease
Lisa Feng, Senior Director, Health Policy, Alexion – AstraZeneca Rare Disease
Holly Fernandez Lynch, Department of Medical Ethics & Health Policy, University of Pennsylvania
Nasha Fitter, VP Patient Network & Data, and Co-Founder & CEO, FOXG1 Research Foundation, Invitae
Julie Fleshman, President And Chief Executive Officer, Pancreatic Cancer Action Network
Mark Fleury, Principal, Policy Development - Emerging Science, American Cancer Society Action Network
Max Flurie, Senior Data Scientist, Trend
Sylvain Forget, Chief Executive Officer, BlueDil International
Miranda Franco, Senior Policy Advisor, Holland & Knight
Albert Freedman, Rare Father/Counseling Psychologist, rarecounseling.com
Neha Gandhi, Head of Global Patient Advocacy and Engagment, Ascendis Pharma
Nicolas Garnier, Senior Director Patient Advocacy Lead, Pfizer
Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)
Laura Genoves, TREND Community
Michele Gerber, Chief Medical Officer, Myeloid Therapeutics
Diego Fernando Gil Cardozo, Executive Director, Federación Colombiana de Enfermedades Raras -FECOER-
Robert Goldstein, Associate Director, Global Patient Advocacy, Neuromuscular, Biogen
Mari Gonzalez, Community Member, TREND Community
Mike Graglia, Founder & CEO, Syngap Research Fund
Alicia Granados, Global Head of Rare Disease Medical Scientific Advocacy & Insights, Sanofi
Frederieke Grevers, Medical Operations Manager, myTomorrows
Erin Griner, Assistant Director of Clinical Research Methodology, Worldwide Clinical Trials
Amy Grover, Director of Patient Advocacy, Catalyst Pharmaceutical Partners
Maria Hadjidemetriou, Specialist Management Account, Medscheme Holdings (Pty) Ltd
Alix Hall, Patient Advocacy Manager, Bionical Emas
Alaa Hamed, Global Head of Medical Affairs Rare Diseases, Sanofi
Nicolas HANSL, ILAPO - Key Account Manager Clinical Trials and EAP, EUROPACCESS
Bree Harlin, Chief Clinical Officer, TC Biopharm
Molly Harper, Chief Business Officer, Synlogic
Rachel Harrison, Associate Director, Early Access Programs, Apellis Pharmaceuticals, Inc
Scott Harwood, Director, Check Rare
Lauren Heiser, Business Development Director, Clinigen Group
Terry Hermiston, Chief Executive Officer, Coagulant Therapeutics
Sharon Hesterlee, Chief Research Officer, MDA
Kenneth Hobby, President, Cure SMA
Karin Hoelzer, Director of Policy and Regulatory Affairs, National Organization for Rare Disorders
Lori Irvin, NICU RN and Nurse Innovator, Children's National
Dwain Irvin, Chief Executive Officer, NovAccess Global, Inc
Stephen Kagan, Global Medical Lead, Genetic Medicine and Pipeline, Rare Disease, Pfizer
Judith Kelleher-Andersson, President and Chief Executive Officer-Neuronascent, NeuroNascent Inc
Ken Kengatharan, Managing General Partner, Atheneos Ventures
Annie Kennedy, Chief of Policy, Advocacy, & Patient Engagement,, EveryLife Foundation for Rare Diseases
Ameet Khara, Head, Patient Advocacy, Scholar Rock
Mark Kiel, Chief Scientific Officer, Genomenon
Maria Kirsch, General Manager, Patient Services, EVERSANA
Terri Klein, President And Chief Executive Officer, National M.P.S. Society
Yishai Knobel, CEO, RxWare
David Knouft, Senior Vice President, Market Access and Field Commercial, Harmony Biosciences, LLC
Akihiro Ko, Chief Executive Officer, Elixirgen Therapeutics, Inc.
Bryan Kobel, Chief Executive Officer, TC Biopharm
Augusta Elizabeth Koroma, Chief Executive Officer, Sickle cell Intervention U.K
Ivan Kouchlev, Principal, IPM.ai
Kollet Koulianos, Senior Payer/Provider Consultant, The National Hemophilia Foundation
Walter Kowtoniuk, Venture Partner, Third Rock Ventures, LLC
Henrik Krook, Vice President Of Commercial Operations, Egetis Therapeutics AB
Sanath Kumar Ramesh, Chief Executive Officer, OpenTreatments Foundation
Shira Landskroner-Eiger, Director, External Innovation Rare Disease, IPSEN
Larissa Lapteva, Associate Director, FDA
Cath Lawrence, VP, Rare Disease, Bionical Emas
Del Lebel, Head, US Government Affairs and Policy, Alexion – AstraZeneca Rare Disease
Nirosha Lederer, Head, US Government, Aetion
Connie Lee, President And Chief Executive Officer, Alliance to Cure Cavernous Malformations
Jeremy Levin, Chairman and Chief Executive Officer, Chairman, BIO, Ovid Therapeutics
William Lin, Program Director, GARDaccess, Partnership for Quality Medical Donations (PQMD)
Jeff Livingstone, Chief Executive Officer, Igia Pharmaceuticals
John Lo, Chief Commercial Officer, Beam Therapeutics
Brett Logan, Director of US Market Access & State Government Relations, Alliance for Regenerative Medicine
Robert Long, Executive Director, Uplifting Athletes
Michael Lynch, Head of Regulatory Affairs CMC, Regulatory Operations and Regulatory Operational Excellence, Alexion – AstraZeneca Rare Disease
Richa Madan Lomash, Scientific Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, NCATS, NIH
Amanda Malakoff, Executive Director, Rare Disease Company Coalition
Manoj Malhotra, Chief Medical Officer, Ovid Therapeutics
Mark Manfredi, Chief Executive Officer, Ikena Oncology
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Silas Martin, Senior Director, Market Access Scientific And External Strategy, Johnson & Johnson
Paolo Martini, Chief Scientific Officer Rare Diseases, Hematology and External R&D, Moderna, Inc.
Kristin Marvin-Keller, Managing Director, H4B-Boston
An Massaro, Lead Neonatologist, Neonatology Program, Office of Pediatric Therapeutics, FDA
Annette Maughan, Co-Founder And Chief Executive Officer, KBG Foundation
Cara Mayfield, VP, Patient Advocacy and Corporate Communications, Homology Medicines
Maria Mccaffrey, Vice President Country Regulatory and Quality, Alexion – AstraZeneca Rare Disease
Cate McCanless, Vice President Of External Affairs, Harmony Biosciences, LLC
Reenie McCarthy, Chief Executive Officer, Stealth BioTherapeutics
Kim McClellan, President, Recurrent Respiratory Papillomatosis Foundation
Susan Mccune, Vice President, Pediatrics & Clinical Pharmacology, thermo fisher scientific
Kristin McKay, Executive Director, Project Alive -
John Mckew, CSO, Lumos Pharma
Jennifer McNary, Founder, One Rare
Christine McSherry, Executive Director, Strategic Collaborations, The Emmes Company, LLC
Zdravka Medarova, Founder, Chief Technology Officer, TransCode Therapeutics
Jen Melanson, Patient Advocate and Caregiver, Rare Disease
Giancarlo Mennella, Managing Director, Egamid Ltd.
Ann-Lise Mikolajczak, Manager Global Health and Access, IFPMA
Nicole Miller, Vice President, Molecular Diagnostics, Global Medical Affairs, Ultragenyx
Samarendra Mohanty, Founder-President, Chief Scientific Officer, Nanoscope Therapeutics
Mara Molinello, Senior Vice President, Head of US Commercial, Alnylam Pharmaceuticals
Mona Moonis, NA Brand Lead, Rare Disease, Kyowa Kirin
Joyce Moore, Executive Director, Patient Recruitment Strategies, Allucent
Kimberly Moran, Head of Rare Diseases, US, UCB
Meghan Morgan-Smith, Associate Director of Therapeutic Expertise Center for Rare Diseases, ICON
Maria-Cruz Morillo, TA Head of Rare Disease, Allucent
Mary Morlino, Patient Services Manager, Global Genes
Claire Murphy, General Manager - Life Sciences US, Terrapinn
Joseph Musumeci, President, BluePrint Orphan
Michael Nace, Brand Director, Rare Disease Advisor
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
George Naumov, Chief Business Officer, RS Oncology
Adora Ndu, Chief Regulatory and interim Legal Officer, BridgeBio Gene Therapy
Catherine Nester, Senior Vice President HCP and Patient Engagement, Inozyme Pharma
Galina Nesterova, Executive Medical Director- Rare Diseases & Pediatrics, PPD, part of Thermo Fisher Scientific
Victoria Niklas, Chief Medical Officer, Oak Hill Bio
Megan O'Boyle, Patient Engagement Lead, Global Genes, RARE-X
Phillip Okwo, Patient, Sickle Beta Zero Thalassemia
Curtis Oltmans, Chief Legal Officer, Fulcrum
Elizabeth Ottinger, Project Management Group Leader, NIH/NCATS
Lisa Padilla, Chief Executive Officer, Beryl Therapeutics Inc
Jennifer Panagoulias, Chief Regulatory and Compliance Officer, Mahzi Therapeutics
Maria Pandolfo, Senior Vice President, Patient Services, Catalyst Pharmaceutical Partners
Kinnari Patel, President & COO, Rocket Pharma
Alana Paull, Director of Associate, Genentech
Melissa Penn, Director Patient Engagement Research & Development, Bayer
Marissa Penrod, Founder and CEO, Rare Disease Advocate, Mom to son with Duchenne Muscular Dystrophy, Team Joseph
Sue Peschin, President and Chief Executive Officer, Alliance for Aging Research
Steven Peskin, President and Chief Executive Officer, Formerly Executive Medical Director Population Health at Horizon Blue Cross Blue Shield o, SRP Advisors, LLC
Wyndi Phillips, VP, Project Management, WEP Clinical
Maria Picone, Chief Executive Officer, TREND Community
Peter Pitts, President, C.M.P.I
Tobias Polak, Director Real-World Data, myTomorrows
Christopher Porter, Vice President, Government Affairs and Policy, Travere Therapeutics
Christopher Porter, Vice President, Government Affairs and Policy, Travere Therapeutics
Stuart Portman, Health Policy Advisor, United States Senate Committee On Finance
Joshua Prabhakar, Vice President, Novartis Gene Therapies
Damon Race, President & Chief Executive Officer, GeneVentiv Therapeutics, Inc
Rachel Radomski, Senior Director & Head, Patient Advocacy, Harmony Biosciences
Paula Ragan, President and Chief Executive Officer, X4 Pharmaceuticals
Harpreet Ram, Senior Advisor and Subject Matter Expert, GARDaccess,, Partnership for Quality Medical Donations (PQMD)
Amy Raymond, Senior Director, Therapeutic Strategy Lead, Rare Diseases, Worldwide Clinical Trials
Debra Regier, Chief, Genetics and Metabolism, Children's National Hospital
Frank Rivera, President, Stronger Than Sarcoidosis
Noreen Rizvi, Principal, Third Rock Ventures
Derek Robertson, President, Maryland Sickle Cell Disease Association
Priscilla Rodriguez, Diversity Inclusion Advocacy Manager, EveryLife Foundation for Rare Diseases
Heidi Ross, Vice President of Policy and Regulatory Affairs, National Organization for Rare Disorders
Jean-Pascal Roussy, Head, Global Public Affairs, Rare Diseases and Rare Blood Disorders, Sanofi
Kacper Rucinski, Delegate, SMA Europe / Founder, Member of Strategy Board, SMA Foundation Poland, SMA Europe (Germany) / SMA Foundation (Poland)
Christopher Rudolf, Chief Executive Officer and Founder, volv global
Pierantonio Russo, Chief Medical Officer, Data and Analytics,, EVERSANA
Kareem Saad, Chief Transformation Officer, GeneDx
Peter Saltonstall, Chief Executive Officer, National Organization for Rare Disorders (NORD)
Sandhya Sanduja, Branch Chief, Pharmacology/Toxicology Branch 2, Office of Therapeutic Products, FDA
Sergio Santillana, Chief Medical Officer, Ikena Oncology
Aaron Schuchart, CBO, Lumos Pharma
Jonathan Schwartz, Chief Gene Therapy Officer, Rocket Pharma
Denise Scots Knight, Chief Executive Officer, Mereo Biopharma
Simone Seiter, Senior Partner, Simon-Kucher
Albert Seymour, President & Chief Executive Officer, Homology Medicines
Christy Shafer, Chief Commercial Officer, Marinus Pharmaceuticals
Nilomi Shah, Director of Medical Affairs Lead, Lexeo Therapeutics
Pallavi Sharma, Senior Director, Scientific Program Management, Adverum Biotechnologies
Conor Sheehey, Health Policy Advisor, United States Senate
Jerry Shen, Chief Executive Officer, Co-Founder, AceLink Therapeutics, Inc
Tricha Shivas, Chief Strategy Officer, Foundation for Sarcoidosis Research
Wen Chyi Shyu, Chief Executive Officer, BRIM Biotechnology, Inc.
Sean Sigmon, VP Business Development, InformedDNA
Dylan Simon, Director of Policy, EveryLife Foundation for Rare Diseases
Amy Simon, Chief Medical Officer, Beam Therapeutics
Amanda Singleton, Director, Biopharma and Strategic Partnerships, GeneDx
Nick Sireau, Chair and Chief Executive Officer, AKU Society
Jayson Slotnik, Partner, Health Policy Strategies, LLC
Tom Smith, Strategic Initiatives Director, Durbin
Rorey Smith, Community Member, TREND Community
Rachel Smith, Executive Director of Rare Disease, Parexel International
Kim Smith-Whitley, Site Lead, Advisor, Scientific and Clinical Affairs, Pfizer
Scott Snyder, Chief Digital Officer, EVERSANA
Marcio Souza, President and Chief Executive Officer, Praxis Precision Medicines
Rodica Stan, Senior Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, NCATS, NIH/NCATS
Kim Stephens, Executive Director, Muenzer MPS Research & Treatment Center
Dean Suhr, President, MLD Foundation
Jamie Sullivan, Director of Public Policy, EveryLife Foundation for Rare Diseases
Marshall Summar, Director, Rare Disease Institute, Children's National Medical Center
Jason Tardio, Chief Operating Officer, Ovid Therapeutics
Mitra Tavakkoli, Vice President; Clinical Development, 4DMT
Ulrich Thienel, Chief Executive Officer, ReAlta Life Sciences
Corinne TRUFFAULT, INTSEL CHIMOS - CEO, EUROPACCESS
Mark Trusheim, Strategic Director, MIT NEWDIGS
Andrew Udell, President of North America, Commercial, Calliditas Therapeutics
Lori Upham, HIE Program Leader, ReAlta Life Sciences
Jennifer Urwongse, DCT Strategy & Innovation Director, PPD, part of Thermo Fisher Scientific
Vinnie Ustach, Applied Computational Biology Lead, GeneDx
Ben van der Schaaf, Partner, Arthur D. Little
Robert-Jan Van Son, General Manager Germany, BeNeLux and Nordic,, Orphalan
Leon van Wouwe, Clinical Innovation Director, volv global
Atul Varadhachary, Managing Partner, Fannin Innovation Studio
Lavni Varyani, Founder, Pharma BP DMCC
Geeta Vemuri, Managing Partner, Agent Capital
Ernesto Vera Sanchez, VP, Dept. Head GXP Strategy, RDC, Allucent
Raghu Vishwanath, Global Head, Franchise Strategy and Operations, Rare Diseases Specialty Care, Sanofi
Daniel Wainstock, Researcher and Rare Disease Advocate, PUC-Rio
Terence Walts, Chief Executive Officer, Cambium Medical Technologies
Mary Wang, Programme Director, Rare Diseases International
Jingsong Wang, President & Chief Executive Officer, Harbour Biomed
Amanda Wang, Vice President Oncology Precision Medicine Franchise Lead For Immuno-Oncology, Bayer Pharma AG
Laurent WARTELLE, INTSEL CHIMOS – Business Development Manager, EUROPACCESS
Yael Weiss, Chief Executive Officer, Mahzi Therapeutics
Steve Winitsky, Vice President, Technical,, Parexel
Ryan Witt, Chief Business Officer, Spinogenix
Kristin Viswanathan Wolff, Senior Director, Global Government Affairs And Public Policy, bluebird bio
Durhane Wong-Rieger, President, CEO & Chair, Canadian Organisation for Rare Diseases & Rare Diseases International
Teonna Woolford, Chief Executive Officer, Sickle Cell Reproductive Health Education Directive
Chris Wurst, Director, Business Development & Sales Operations, WEP Clinical
Lisa Yañez, Chief Executive Officer, Aerami Therapeutics
Yuliya Yasinskaya, Medical Officer, FDA
David Young, Chief Executive Officer, Processa Pharmaceuticals, Inc.
Jennifer Young, Vice President, Global Policy and Public Affairs - Specialty Care, Pfizer
Christine Yu, Chief Executive Officer, P2P Syncro
Marjorie Zettler, Executive Director of Clinical Science, Regor Pharmaceuticals Inc.
Tara Ziegler, Business Development Manager, GeneDx
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