Allopurinol designated as the first orphan drug for Marfan syndrome treatment

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, a rare connective tissue disease which has no cure to date. This disease causes an aortic aneurysm (an abnormal dilation of the aorta) and affects about 7 in 100,000 people in the European Union. Drugs known as orphan drugs are intended to treat such rare conditions that pharmaceutical companies need favorable conditions to market them.

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