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...

To read the full abstract: Diabetes Care. 2017;40:1034-1040Researchers from The Children’s Hospital at Westmead, Sydney, Australia as well as scientists from institutions from three different continents, America, Europe and Australia have combined their data from large diabetes registries in order to: examine the prevalence of celiac disease (CD) in populations of patients with T1DM; investigat...

Abstract: Lancet. 2019 May 16.In brief: In a randomised, active-controlled, open-label, phase 3 trial, burosumab (an anti-FGF23 antibody) demonstrated significantly greater clinical improvements in rickets severity, growth, and biochemistries among children with X-linked hypophosphataemia compared with continuation of conventional therapy with oral phosphate and active vitamin D ...