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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.
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.
Virus collections are vital resources for understanding the basis of diseases and formulating control strategies. An EU-funded project is developing an easily accessible global archive of pathogens - a way of boosting research into protecting human and animal health.
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.
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.
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.
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.
An EU-funded project has provided new hope for the 2 million people in Europe who suffer from rare kidney diseases thanks to groundbreaking research that has led to diagnostic tests and a new type of treatment.
Most rare diseases are linked to specificities of the patients' genes - but what these are, exactly, remains to be determined for the vast majority of these illnesses. Tackling this challenge, an EU-funded platform is helping to generate answers from as few as two known cases by enabling scientists to pool and compare genomic and clinical data.
EU-funded scientists have conducted groundbreaking research into brain cells that control memory formation and recollection, offering the promise of novel treatments for a potentially fatal disease that has a particularly devastating impact on the lives of children and young adults.
