In Europe, a disease is defined as rare when it affects fewer than one out of every 2,000 people. However, as many as 30 million people may suffer from one of the more than 6,000 existing rare diseases.
This section of the website provides information for companies and individuals involved in developing and marketing medicines for human use in the European Union (EU)
A useful resource of Cellular & Gene Therapy Guidance Documents
Vivet Therapeutics raises €37.5 million in Series A financing round to advance novel gene therapies for treating inherited metabolic diseases