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Advancing therapies for paediatric renal ciliopathies - TheRaCil

We are delighted to be the patient partner in a 4-year research programme funded through Horizon Europe, the European Union's framework program for research and innovation. “Therapies for Renal Ciliopathies” (TheRaCil) was in the call "Development of new effective therapies for rare diseases".

TheRaCiL brings together 16 partners - 15 institutions in 6 countries - 3 European consortia and the Ciliopathy Alliance with the ambition to develop appropriate and targeted treatments for paediatric renal ciliopathies. TheRaCil will benefit from a European Commission funding of 7,425,446 euros and a co-funding of 540,520 euros from UK Research and Innovation (UKRI). 

Development of effective new therapies for rare diseases: the case of renal ciliopathies

Renal ciliopathies are rare genetic diseases caused by dysfunction of the primary cilium, impacting tissue development and balance. Paediatric renal ciliopathies, including nephronophthisis (NPH) and autosomal recessive polycystic kidney disease (ARPKD), contribute to 15% of childhood chronic kidney disease (CKD) and often progress to end-stage kidney disease (ESKD), which poses significant challenges for patients, families, and healthcare systems. TheRaCil aims to address the urgent need for effective treatments for these conditions. TheRaCil project is a multidisciplinary consortium including clinicians, geneticists, biotechnology companies, and experts in complementary research fields such as cilia biology, bioinformatics, data science, and artificial intelligence (AI) along with patient advocacy groups. By leveraging the expertise of this consortia, TheRaCil aims to develop prognostic tools and therapies that can benefit most patients affected by renal ciliopathies.

Read the full press release here.