Alexion Pharmaceuticals Inc. Release: Paediatric Patients With Hypophosphatasia Receiving Investigational Asfotase Alfa Had Sustained Improvements In Growth And Physical Function

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DUBLIN, Ireland--(BUSINESS WIRE)--Alexion Pharmaceuticals today announced that researchers presented data from the open-label extension phases of two Phase 2 studies of asfotase alfa, an investigational enzyme replacement therapy, in paediatric patients with hypophosphatasia (HPP).1,2 In the ongoing studies, researchers observed sustained improvements in growth, strength, physical function, and other key measures in paediatric patients with HPP who were treated with asfotase alfa for up to three years. HPP is a genetic, chronic and progressive ultra-rare metabolic disease that can lead to ongoing damage to multiple vital organs, destruction and deformity of bones, and premature death.3-7 Data were presented today at the 53rd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Dublin, Ireland, following a presentation of similar data earlier this month at the American Society for Bone and Mineral Research (ASBMR) 2014 Annual Meeting.8,9

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