Uncommon solutions for rare diseases
Rare diseases are uncommon, but there are still thousands of different conditions which together affect between 27-36 million people in the EU. This month, Horizon examines the latest efforts to tackle rare diseases as well as new technology to better diagnose uncommon conditions and novel ways to reduce the socioeconomic burden of unusual disorders.
Researchers are working hard to fight childhood dementia and hope a new enzyme replacement therapy can help those unlucky enough to have such a debilitating disease.
Rare muscle diseases have a devastating impact on the affected individual and their families, but 3D-drug screening could lead to better medicines being developed which would also relieve the huge economic toll of their treatment.
A new EU-wide approach to funding rare disease research could help patients secure access to new treatments, says Dr Daria Julkowska, scientific coordinator on rare diseases at the French National Research Agency.