Dutch initiatives in therapy development and access to medicines for patients with rare diseases are joining forces. You are invited to join us on Monday, June 3rd 2024, to help shape this collaboration!

During the meeting, several local plans for therapy development for rare diseases will be discussed, and RARE-NL will be presented. RARE-NL is a national collaborative initiative for rare diseases and drug repurposing involving various university medical centers (UMCs) including Radboudumc (Therapy Accelerator for Rare Diseases), Amsterdam UMC (Medicines for Society), and LUMC (NFKC), aiming to bring medicines to patients in a socially responsible manner.

RARE-NL will become a collaborative hub of the national expertise center for therapy development (FAST). The goal of RARE-NL is to efficiently address case studies and integrate field experiences at the national level: from case to system.

Program for Monday, June 3rdTime: 1:00 PM – 6:00 PM

Location: De Vereeniging, Nijmegen

12:30 – 1:00 PM Arrival with coffee and tea

The Present: Local Initiatives in Therapy Development for Rare Diseases

1:00 – 1:05 PM Opening

Prof. Dr. Dirk Lefeber

1:05 – 1:20 PM Patient Perspective: Living with a Hereditary Disease Without Medication

Jeanette Mens and Prof. Dr. Bart van de Warrenburg

1:20 – 2:00 PM Therapy Accelerator for Rare Diseases – Radboudumc

Prof. Dr. Saskia de Wildt, Dr. Gerty Schreibelt, Dr. Joanna in ‘t Hout, and Dr. Rob ter Heine

2:00 – 2:20 PM Academic Pharma – LUMC

Prof. Dr. Teun van Gelder

2:20 – 2:40 PM Medicines for Society – Amsterdam UMC

Prof. Dr. Carla Hollak

2:45 – 3:15 PM Break

The Future: National Collaboration

3:15 – 3:25 PM Therapy Development from the NFU Perspective

Prof. Dr. Guillén Fernández Reumann

3:25 – 3:35 PM National Collaboration through RARE-NL

Dr. Saco de Visser

3:35 – 5:00 PM Future Exploration

Dr. Saco de Visser

In breakout sessions, attendees will discuss how RARE-NL can support innovation and affordability together. What support do academic researchers/developers need? How do we organize socially responsible investments?

We hope to see you on June 3rd! Together, we make a difference for rare diseases and drug repurposing.

Best regards,

Dirk Lefeber (Therapy Accelerator for Rare Diseases)

Teun van Gelder (NFKC)

Carla Hollak (Medicines for Society)

Saco de Visser (FAST)