PCD-ZON
Working together for better Primary Ciliary Dyskinesia care
Standardized care, Unified research and National registry
Eric Haarman, AUMC
Tamara Paff, UMCU
Josje Altenburg, AUMC
Joël Israëls, AUMC
Evelien van der Hout, UMCU ReCode Therapeutics Domenique Zomer, NCFS Jacqueline Plat, PCD
This project brings together hospitals, researchers, industry and patient organizations to improve care for people with Primary Ciliary Dyskinesia (PCD) in the Netherlands.
PCD is a rare inherited lung disease that affects around 1 in 10,000 people. It can cause chronic coughing, sinus and ear problems, and repeated airway infections. Because these symptoms can be mistaken for other conditions, it may take years before people receive the correct diagnosis. Delayed diagnosis can lead to permanent lung damage and a reduced quality of life.
New RNA-based treatments that target the underlying cause of PCD are now being developed. To ensure that people with PCD in the Netherlands can benefit from these innovations, a strong national infrastructure is needed. The project will establish a national PCD registry to securely collect and connect information from people with PCD and link this data to European initiatives. It will also develop standardized diagnostic and care pathways to support high-quality and consistent care across the country.
In addition, the project will raise awareness of PCD among healthcare professionals to support earlier diagnosis and work with industry to prepare for future clinical trials of new genetic treatments.
Together, Amsterdam UMC, UMC Utrecht, ReCode Therapeutics, the Dutch Cystic Fibrosis Foundation, and the PCD patient organization aim to create a stronger foundation for PCD care and research in the Netherlands.
The project will deliver a national registry, improved care pathways, tools to better understand disease progression and a roadmap for introducing new therapies. Ultimately, this will help improve the lives of people with PCD and prepare the Netherlands for the next generation of treatments.
