News

Written on 26 February 2026.

Strong in Fragility: A Father’s Journey with ARPKD on Rare Disease Day

My name is Andrea. I am 43 years old and the father of three wonderful children: Giuseppe (15), Francesca (10) and Alessandro (5). Two of them, Giuseppe and Alessandro, live with a rare disease: Autosomal Recessive Polycystic Kidney Disease (ARPKD). Being a father in these circumstances means learning very early that life no longer follows the “normal” rhythm of most families. Every decision becomes intertwined with appointments, treatments, hospital stays and waiting times, and the future is measured in small but vital goals.

Written on 08 December 2025.

ARTICYST Survey

The ARTICYST consortium brings together leading European centres of excellence in kidney research, including Translational Nephrology at the Faculty of Medicine and University Hospital Cologne in Germany, the Department of Nephrology at University Medical Center Groningen in the Netherlands, and PKD International representing the patient voice at international level.

Important Update: Novartis Acquisition of Regulus Therapeutics

Novartis has completed its acquisition of Regulus Therapeutics, the biotechnology company developing farabursen, an investigational therapy designed to target microRNA-17 with the aim of slowing cyst growth and preserving kidney function. The transaction has a potential value of up to USD 1.7 billion, reflecting increased commitment to advancing research and development in ADPKD - an area of considerable unmet medical need.

Written on 06 June 2025.

ERA - Advisory Board with Vertex Pharmaceuticals

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.

Written on 13 May 2025.

PICKED & DISCO-I Joint Meeting in Toulouse

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.

Written on 01 May 2025.

Novartis Acquires Regulus Therapeutics for ADPKD Therapy

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.

Written on 10 March 2025.

Members Meeting News

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.