A rare infectious disease that can cause permanent blindness may soon have an approved treatment for the first time. A formulation of the molecule polihexanide, optimised by the EU-funded ODAK project, has been shown to cure 87 % of patients. The drug will soon be available to patients across Europe through an early access programme.
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.
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.
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.
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.
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.
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.
