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). 

Alström Syndrome UK is pleased to join Genetic Alliance UK’s Future for Rare Steering Group. Together, we are taking a leading role in shaping the future of rare conditions policy in the UK.

The Future for Rare survey is designed to capture a comprehensive picture of the current experiences and priorities of the genetic, rare and undiagnosed community across the UK.

We welcome views from everyone, whether you are living with a genetic, rare and undiagnosed condition, caring for a loved one, or working for a support organisation, as a researcher or a healthcare professional. Every perspective is valued equally and will directly inform our recommendations to all four governments.

Share your experiences via the Future for Rare survey.

Please submit your response by Friday 24 April 2026.

For more information on how this information will inform the campaign, please visit the Future for Rare webpage.

Genetic Alliance UK – UK Rare Diseases Framework Consultation

Join us on Thursday, 11th June 2026 from 12 noon to 2 p.m. to help Genetic Alliance develop practical and realistic policies for Rare Diseases to feedback to the UK Government. We want to ensure that the voices of the Ciliopathy community are heard so would welcome, people with lived experience, support groups, researchers and clinicians to join us for this important workshop.

Register here for the meeting

The Ciliopathy Alliance had a great afternoon at the Houses of Parliament as guests of Rare Disease UK where Andrew Stephenson (Minister for Health and Secondary Care) launched the governments plan to continue supporting patients with Rare Diseases.  The standout points were the piloting of Syndromes Without a Name clinics for people with rare undiagnosed conditions and the 'Generation Study' where newborn babies witll be screended for rare diseases. 

It was also a great opportunity for  us to catch up with old friends and colleagues from the Rare Disease community.