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Fabry Disease is a rare genetic lysosomal storage disease resulting from the deficient activity of a different enzyme, alpha-galactosidase A (a-Gal A). The disease affects around one in 40,000 men and one in 60,000 women and causes renal failure and/or cardiac disease, and (usually) early death. 

Pharming believes that its technology platform can produce a very pure, less immunogenetic treatment that will compare favourably with current available therapies on efficacy and ease of administration. 

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