ESPE Yearbook of Paediatric Endocrinology

Abstract: Lancet Diabetes Endocrinol. 2019 Feb;7(2):93–105.In brief: The study reports outcomes of a single-arm 7-year phase 2 extension trial of Asfotase alfa for infants and children with life-threatening hypophosphatasia who received a median of 6·6 years of therapy. The early improvements previously reported were sustained for up to 7 years of treatment.<p class...