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News covering rare disease

News Update: Join Us at the ECRD 2024!


Our Newcastle University Centre for Rare Disease is proud to be an associate partner at the European Conference on Rare Diseases & Orphan Products (ECRD) 2024. Join us for this major event on May 15-16, 2024, available online through a fully interactive platform. The ECRD is the largest, patient-led, rare disease policy-shaping event held in Europe. With over 1000 participants, the Conference is an unrivalled opportunity to network and exchange invaluable insights within the rare disease community.


Discussions will be taking place on areas including breakthrough therapies funding, mental health support for rare disease patients, equitable diagnosis strategies, access to specialised care, and robust national action plans for rare diseases.  The conference, organised under the auspices of the Belgian Presidency, will culminate in the output of a co-created, open letter to EU Institutions and country leaders, providing a solid legacy for the future of the newly elected EU leadership.  


New £12 million research centre to improve studies of rare disease

A new £12 million research centre has been launched to improve clinical trials for patients with rare diseases.

Honorary lecturer wins Researcher of the Year award

The Newcastle University lecturer was presented with a national award for his innovation in surgery during an awards ceremony in Leeds.

£30M study paves the way for new treatments for people with cirrhosis

The most extensive clinical study into liver cirrhosis ever conducted worldwide has been announced.

White paper on referral pathways for gene therapy

As gene therapy to treat Duchenne muscular dystrophy is approved in America, a report highlights how NHS referral for gene therapy trials, and approved treatment, need to be agreed.

Pioneering treatment highly effective for rare kidney disease

A pioneering drug for a rare kidney disease prevents organ failure and significantly improves the outcome for patients, new research has confirmed.