Alexion reports new data that show that children with perinatal- and infantile-onset hypophosphatasia (HPP) treated with Strensiq® (asfotase alfa) had statistically significant improvements in bone healing as assessed radiographically, which were sustained through 3.5 years of treatment … more

Separately, Alexion said that Japan has approved Kanuma® (sebelipase alfa) for the treatment of patients of all ages in Japan with lysosomal acid lipase deficiency (LAL-D). Kanuma is the first therapy approved in Japan for the treatment of patients with LAL-D, a genetic and progressive ultra-rare metabolic disease in which patients suffer multi-organ damage and premature death … more