Featuring Expert Speakers
Orphan Drugs & Rare Diseases Global Congress 2022 Europe
- Doug Danison, Head of Europe, bluebird bio
- Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck
- Helena Baker RGN, CEO, Rare Disease Nurse Network
- Emily Crossley, Co-founder, Duchenne UK
- Nick Sireau, PhD, Chair and CEO, AKU Society
- Owen Marks, Country Head for Rare Diseases Unit, Pfizer UK*
- Femida Gwadry-Sridhar, CEO and Founder, Pulse Infoframe Inc.
- Senior Representative, Rare Diseases Unit, Pfizer, UK
- Lesha D. Shah, MD, Medical Director, Icahn School of Medicine, Mount Sinai
- Philippa Norman, Projects Coordinator, Findacure
- Elisabetta Zanon, Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine
- Josie Godfrey, Director, JG Zebra Consulting
- Lindsay Weaver, Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases
- Christian Girard, Strategic Consultant, Orphan Drugs Industry Advisory & Intelligence
- Senior representative, Illingworth Research Group, a Syneos Health Company
- Sophie Laribiere, Sr Director, Global Regulatory Affairs, Alexion Pharmaceuticals
- Rhian Kiely, RWE Commercial Director, Europe, Open Health Evidence & Access
- Martina Garau , Director, Office of Health Economics
- Andrew Kim, VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines
- Ryotaro Ishikawa, Global Market Access Director, Novartis
- Simu Thomas PhD, Vice President, Global Head HEOR, Alexion Pharmaceuticals
- Keith White, Head, Global Market Access, Amylyx Pharmaceuticals
- Arvin M. Gouw, Ph.D., VP of Research & Development, Rare Genomics Institute
- Angela Columbano, Head Business Development & Partnership, Genethon
- Paul Beresford, International Biotech Executive
- Saira A. Jan, VP & Chief Pharmacy Officer, Horizon Blue Cross Blue Shield of New Jersey
- Paolo Morgese, EU Director Market Access, Alliance for Regenerative Medicine
- Manolo Bellotto, PhD, President, Gain Therapeutics SA
- Alexander Natz, LL.M., Secretary-General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
- Karen Matthews, Business Development Manager, National Institute for Health Research*
- Kerry Leeson-Beevers, National Development Manager & Project Lead, Alström Syndrome*
- Senior Representative, National Institue for Health and Care Excellence (NICE)*
- Paul Greenland, President EMEA, Amryt Pharma
- Simon Jones, MD, Medical Director, NIHR Clinical Research Facility, NIHR
- Sheela Upadhyaya, Associate Director - Highly Specialised Technologies, NICE
- Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
- David Rose, BD, RARE Revolution Magazine, Ultra-Rare Disease Patient Speaker
- Karen Matthews, Business Development Manager, CRNCC, NIHR Clinical Research Network
- Eric Alton, Professor, Imperial College London, Head, UK CF Gene Therapy Consortium
- Joe Brice, Head, Franchise Public Affairs, EUCAN, Takeda*
- Farhat Khanim MD, Director of Research, Institute of Clinical Sciences, University of Birmingham
- Zizi Imatorbhebhe, MBA MS PMP® Senior Vice President, Global Strategy & Development, Rare Disease, Ergomed
- Daniel Lewi, Head of Business Development and Patient Advocacy, Pulse Infoframe Inc.
- David Ross, Patient Advocate, Rare Disease Mental Health
- Peter MacLennan, CEO, Tailored Clinical Research Solutions Ltd
- Juliet Hulse, Sr. Director, Global Research Nurse Strategy, Illingworth Research Group
- Carole Scrafton, CEO & Co-Founder, FibroFlutters
- And many more...

Alexander Natz, LL.M.
Secretary-General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)

Keith White
Head, Global Market Access, Amylyx Pharmaceuticals

Elisabetta Zanon
Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine

Andrew Kim
VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines

Robert Donnell
Executive Vice President – Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

Angela Columbano
Head Business Development and Partnership, Genethon

Helena Baker RGN
CEO, Rare Disease Nurse Network

Simu Thomas PhD
Vice President, Global Head HEOR, Alexion Pharmaceuticals

Emily Crossley
Co-founder and joint CEO, Duchenne UK

Lindsay Weaver
Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases

Philippa Norman
Projects Coordinator, Findacure

Eric Alton FRCP, FMedSci,
Professor of Gene Therapy and Respiratory Medicine, Imperial College, London

Paul Beresford
International Biotech Executive

Josie Godfrey
Director, JG Zebra Consulting, Project Director, Duchenne UK's Project Hercules

Doug Danison
Head of Europe, bluebird bio

Christian Girard
EU Business Operations Consultant, Acer Therapeutics

Ramona Reichenbach
Head Managed Access Center of Excellence (MACoE), Novartis

Dr Femida Gwadry-Sridhar
Chief Executive Officer and Founder, Pulse Infoframe

Ryotaro Ishikawa
Global Market Access Director, Novartis

Martina Garau
Director, Office of Health Economics

Saira A. Jan
VP & Chief Pharmacy Officer, Horizon Blue Cross Blue Shield of New Jersey

Manolo Bellotto, PhD
President & General Manager, Gain Therapeutics

Owen Marks
Country Head for Rare Diseases Unit, Pfizer UK

Lesha D. Shah, MD
Assistant Professor of Psychiatry, Medical Director, Child, Adolescent and Family Services, Icahn School of Medicine, Mount Sinai

Paul Greenland
President EMEA, Amryt Pharma

Pedro Franco
Director for Global Regulatory & Scientific Policy (GRASP), Merck

Farhat Khanim MD
Director of Research, Institute of Clinical Sciences, University of Birmingham

Karen Matthews PhD
Business Development Manager, NIHR

David Edward Rose
Ultra-Rare Disease Speaker (1 in 300 million), Business Development & Sales, Rare Revolution Magazine

Eugean Jiwanmall,
Sr Research Analyst, Medical Policy & Technology Evaluation, Independence Blue Cross

Paolo Morgese,
EU Director Market Access, Alliance for Regenerative Medicine

Sheela Upadhyaya
Associate Director - Highly Specialised Technologies, NICE

Rhian Kiely
RWE Commercial Director, Europe, Open Health Evidence & Access

Senior Representative
Illingworth Research Group

Pedro Franco
Director for Global Regulatory & Scientific Policy (GRASP), Merck

Dr. Nicolas Sireau
CEO and Chair of Trustees, AKU Society

Robert Donnell
Executive Vice President – Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

Zizi Imatorbhebhe, MBA MS PMP®
Senior Vice President, Global Strategy & Development, Rare Disease, Ergomed

Andrew Kim
VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines

Elisabetta Zanon
Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine

Arvin M. Gouw, Ph.D.
VP of Research & Development, Rare Genomics Institute

Peter MacLennan
CEO, Tailored Clinical Research Solutions Ltd

Juliet Hulse
Sr. Director, Global Research Nurse Strategy, Illingworth Research Group

Daniel Lewi
Head of Business Development and Patient Advocacy, Pulse Infoframe Inc.

David Ross
Patient Advocate, Rare Disease Mental Health

Carole Scrafton
CEO & Co-Founder, FibroFlutters

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