Success Stories

Replacing an enzyme to control a very rare disease

Until recently, there was no treatment specific to alpha-mannosidosis, one of the many rare diseases that jointly affect some 30 million citizens in Europe alone. Today, there is as EU-funded research developed enzyme-replacement therapy to stop the illness in its tracks, and this medicine is on the market.

Published: 12 October 2020

Insights into treating rare cancer tumours benefit patients

What is the best way to tackle sarcomas? Although clinical trials help to generate insight, they are hard to set up for rare diseases. An EU-funded project has organised several trials focusing on these malignancies, providing insight that is already helping to save more lives. It has also paved the way for further research to help patients.

Published: 7 September 2020

Faster, more affordable vaccine purification

Generating safe antigens to induce immunity is one of several key steps in vaccine production. Purification is another, and it is typically a complex process. EU-funded researchers have designed a way to simplify the process, boost yields, limit waste and reduce costs, in a bid to help make more doses available more affordably.

Published: 7 May 2020

Faster rare anaemia research

An EU-funded project has developed a blood-cell analysis device that helps doctors and scientists better understand the causes and mechanics of rare forms of anaemia - potentially speeding up the development of new treatments adjusted to the needs of patients with these diseases.

Published: 25 April 2019

Viral gene therapy could treat incurable disease

EU-funded researchers have conducted the first successful trials of an innovative gene therapy to treat a rare and debilitating metabolic disorder, using a small and innocuous virus as a carrier for genetic information to correct the functioning of liver and muscle cells.

Published: 3 April 2019

Gene therapy trials point to cure for rare liver disease

EU-funded researchers are close to developing a cure for Crigler-Najjar syndrome, an extremely rare, life-threatening liver disease. The groundbreaking research, currently being validated in clinical trials, could also pave the way for the treatment of other genetic disorders.

Published: 27 February 2019

Fast, effective treatment for autoimmune rheumatic diseases

Autoimmune rheumatic diseases like rheumatoid arthritis, lupus and scleroderma are debilitating and occasionally life-threatening. An EU and industry-funded project aims to improve treatment by getting the right therapies to the right patients, fast.

Published: 12 October 2018

Better management and treatment of neurological diseases

Management and treatment of neurological diseases is one of the biggest challenges facing medicine today. EU-funded researchers are developing computational tools to improve understanding of how such diseases progress as a means to help treat patients.

Published: 22 June 2018

New approach to advance drug pipeline for cystic fibrosis

For patients with rare genetic mutations of the debilitating disease cystic fibrosis, there are few options for effective treatment. EU-funded researchers are working to meet their needs, using new developments in personalised medicine to advance the testing of novel and promising drugs.

Published: 19 June 2018

Pooling resources to make better diagnoses of rare diseases

By pooling patient data and applying state-of-the art genetic methods, EU-funded research is improving the diagnosis of rare diseases that affect the lives of tens of millions of EU citizens.

Published: 12 June 2018