27 October 2025 | Workshop Day
28-29 October 2025 | Conference & Exhibition
RAI Congress Centre, Amsterdam
The World Orphan Drug Congress is the largest and most established orphan drug & rare disease meeting of its kind across the globe.
From cell and gene therapy, genetic testing, and market access, to real world evidence, this one meeting covers the whole orphan drugs value chain where science, government and manufacturers all come together to create groundbreaking progress.
Attendees
Speakers
Exhibitors
Days
1 to 1 Partnering Meetings
Networking Connections
Covering cell & gene therapy, genetic testing, market access, real world evidence, orphan drugs policy and much more
130+ leading solution providers from across the globe, featuring new ways of thinking and novel partnerships to generate solutions.
Networking is at the heart of World Orphan Drug Congress. Our congress allows you to connect directly with thousands of pharma experts across 3 days of built-in 1-2-1 networking
Elizabeth de Lange, PhD, is Professor of Predictive Pharmacology at the Division of Systems Pharmacology and Pharmacy of the LACDR (Leiden University). Her group develops predictive (CNS) pharmacokinetic (PK)- pharmacodynamic (PD) relationships in human, in health and disease, mainly using “smart” ...
Alicia Granados is a highly accomplished leader in the field of rare diseases, currently serving as the Head of Global Rare Diseases Medical Scientific Advocacy at Sanofi, where previously she headed its global HTA strategy .With almost 15 years of experience in the pharmaceutical industry, Alicia...
Aurora Mateos is Founder and General Director of Menkes International (MIa), a charity focused on fighting the rare disease called Menkes. She is an international lawyer (she has been Consultant to United Nations for more than fiftheen years) on environmental law, specially law of the sea, as well...
Tara Smith is the Executive Vice President, Innovative Therapies at Med-Life Discoveries LP, a North American company developing a first-in-class plasmalogen metabolic replacement treatment. Their current focus is the ultra-rare disease Rhizomelic chondrodysplasia punctata (RCDP). Tara has over 15...
Andy Barrick- CEO Multiple System Atrophy (MSA) Trust UK Andy Barrick has worked in the UK voluntary sector for over 30 years, the last ten with the MSA Trust. Since 2015 the MSA Trust has doubled the number of people they support and have prioritised engagement with our members. Andy has overseen ...
Dr. Juliane Bernholz is an experienced CEO, cell biologist, and molecular geneticist with over 30 years of leadership experience in the pharmaceutical industry having worked and lived in the US and multiple countries in Europe. Currently, she serves as the CEO of AM-Pharma, a private clinical stage ...
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