For example, the prevalence of the mucopolysaccharidoses group of metabolic disorders is 14 times higher in Saudi Arabia than in the United States, providing a significant market opportunity.

Martin Mackay, head of R&D at Alexion, talks to pharmaphorum about the company's research pipeline for severe and ultra-rare diseases. Alexion has become hugely successful in recent years thanks ...

### 2.1 - Primary Congenital Forms/Childhood Glaucomas Primary congenital glaucoma is a rare disease but has a major impact on the child’s development and quality of life over his/her whole life span.