News

PKD International participated in the ADPKD Community Advisory Board organized by Vertex Pharmaceuticals. Members from Spain, Germany, Italy, and the UK brought the voice of the ADPKD patient community from across Europe to the table.

The PICKED & DISCO-I Joint Meeting will take place from May 13-15, 2025, at the Institut des Maladies Métaboliques et Cardiovasculaires (I2MC) in Toulouse, France. This three-day event brings together doctoral candidates, leading experts, and distinguished speakers from across Europe to foster collaboration, share knowledge, and advance research in kidney disease and innovative omics technologies.

Novartis has announced an agreement to acquire Regulus Therapeutics, a San Diego-based clinical-stage biopharmaceutical company focused on developing microRNA therapeutics. Regulus' lead asset, farabursen, is a potential first-in-class, next-generation oligonucleotide targeting miR-17 for treatment of autosomal dominant polycystic kidney disease (ADPKD).

The transaction includes USD 0.8 billion upfront with a potential additional USD 0.9 billion payment upon achievement of future regulatory milestones. The transaction is expected to close in the second half of 2025.

Meeting Summary

Welcome & Introduction: Flavia Galletti outlined objectives covering survey insights, financial updates, communication tools, and collaborations.

Member Survey Insights: Members identified a need for better communication, financial transparency, and strategic partnerships, with suggestions to enhance digital efforts and member collaboration.

Financial Overview & Membership Fees: PKD International holds approximately 26,000 CHF (approximately 27,000 EUR). A proposal was made to hire a project manager and communication agency, with discussion on membership fees and alternative funding sources.

Are Your Kidneys OK? Detect early, protect kidney health

Chronic kidney disease (CKD) is estimated to affect approximately 850 million people worldwide. If left undetected and not treated timely, CKD can progress to kidney failure, leading to severe complications and premature mortality. By 2040, CKD is projected to become the 5th leading cause of years of life lost, highlighting the urgent need for global strategies to combat kidney disease.

Populations at high risk for kidney disease should undergo targeted testing.

Care Pathways Toolkit (JARDIN)

Designed to support healthcare professionals, project managers, and patient representatives in co-developing ERN care pathways, this toolkit proposes a model that ensures the needs and priorities of People Living With Rare Diseases (PLWRD) are embedded in the resulting pathways.