23rd Orphan Drugs and Rare Diseases Global Congress 2025 Europe
Addressing the challenges in drug development, treatment availability, and access.
Copthorne Tara Hotel London Kensington, London, UK, London, United Kingdom
Wednesday 19th - Thursday 20th March 2025
KEY INDUSTRY EXPERT SPEAKERS
- Prof James LEVINE MD PhD, President, Fondation Ipsen
- Dr Peter Davis, Chair, British Paediatric Surveillance Unit Scientific Committee
- Amanda Bok, Chief Partnership Officer, The Synergist
- Alberto Malerba, Lead, Gene Medicine Laboratory for Rare Diseases, Royal Holloway University
- Nick Sireau, CEO and Chair of Trustees, AKU Society
- Prof Guillaume Canaud, Translational Medicine & Targeted Therapies Unit, Necker Enfants Malades
- Lindsay Randall, Mother, Founder, CEO, Arthur’s Quest
- Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.
- Dan Lewi, Head of Business Development and Patient Advocacy , Pulse infoframe Inc.
- Sheela Upadhyaya, Chair Elect, Rare Disease Special Interest Group, ISPOR
- Carole Scrafton, Director & Co-Founder, Flutters and Strutters
- Josie Godfrey, Co-Founder and CEO, Realise Advocacy
- Nicholas Brooke, Founder & Executive Director, Synergist
- Lindsay Birrell, Co-CEO, Realise Advocacy
- Sue Verma, Chief Medical Officer, Leadiant Biosciences
- Cpt. Luke Rosen, Founder, Rescue7: Firefighters for Patients, and KIF1A.ORG.
- Dr Pooja Takhar, Joint Chief Executive, Tuberous Sclerosis Association
- Daniel O’Connor, Director, Regulatory Policy and Early Access, ABPI
- Francis Pang, SVP Global Market Access and Geographic Expansion, Orchard Therapeutics
- Dr. Rüdiger Schulze, Vice President and General Manager, DACH, Ultragenyx
- Robert Mitchell-Thain, CEO, PBC Foundation
- Anna Kole, Global Patient Engagement Lead - MG, UCB
- Enrico Piccinini, Head of Europe & International, Rare Diseases, Chiesi Group
- Flaminia Macchia, Head, Global Rare Disease Policy, AstraZeneca
- Alicia Granados, Global Head Scientific Advocacy & Insights, Rare Diseases, Sanofi
- Manel Cascallo, General Director, Theriva Biologics Inc
- Dr Lucy McKay, Chief Executive Officer, Medics4RareDiseases
- Alexander Natz, Secretary General, EUCOPE
- Jennifer Schranz, SVP, Global Head of Rare Diseases, Ipsen
Prof James LEVINE
President
SURABHI VERMA
Chief Medical Officer
Amanda Bok
Chief Partnership Officer,
Sheela Upadhyaya
Chair Elect, Rare Disease Special Interest Group
Nick Sireau
Chair & CEO
DAN LEWI
Head of Business Development and Patient Advocacy
Pulse infoframe Inc., Co- founder, CATS Foundation
DAN LEWI
Head of Business Development and Patient Advocacy
Prof Guillaume Canaud
Translational Medicine & Targeted Therapies Unit
Dr Alberto Malerba
Lecturer in Gene therapy, Gene Medicine Laboratory for Rare Diseases –
Malerba Research Group, Department of Biological Sciences Royal Holloway, University of London
Dr Alberto Malerba
Lecturer in Gene therapy, Gene Medicine Laboratory for Rare Diseases –
Dr Peter Davis
Chair
Dr. Femida Gwadry-Sridhar
Founder and CEO
Alexander Natz
Secretary General
CAROLE SCRAFTON
Director & Co-Founder
Cpt. Luke Rosen
Founder
DR USMAN KHAN
Chair
Bob Stevens
Group CEO, MPS Society, Vice Chair
DR POOJA TAKHAR
Joint Chief Executive
DR. RÜDIGER SCHULZE
Vice President and General Manager
ROBERT MITCHELL-THAIN
CEO
ANTHONY HALL
Co-Founder, Member of Board & Scientific Advisor
STEFANO BENVENUTI
Head of Public Affairs and Market Access