Following the positive results of a four year clinical trial of nitisinone in AKU that was funded by the European Commission.
To further encourage the development of treatments for rare diseases, EMA waives all fees for scientific advice for academia developing orphan medicines
Nature Medicine has published the first results of a gene therapy based clinical trial with patients suffering from Fanconi anaemia.
Research and innovation - News alert
A four-year clinical trial of nitisinone for treating the rare disease alkaptonuria (AKU), has been analysed and shown positive results