Searchable abstracts of presentations at key conferences in endocrinology

ea0049gs1.4 | Guided session 1 | ECE2017

Cardiovascular health issues in turner syndrome

Backeljauw Philippe

Individuals with Turner syndrome (TS) have an increased risk for congenital and acquired cardiovascular disease (23–50%). This results in increased morbidity and mortality throughout the TS lifespan. There is an increased prevalence of bicuspid aortic valve malformation, coarctatio of the aorta, elongation of the transverse aortic arch, and partial anomalous venous return. In addition, TS females may develop aortopathy that may result in clinically significant aortic dila...

ea0049gp66 | Developmental & Protein Endocrinology | ECE2017

Achievement of a suitable basis of comparison in phase 2 and 3 clinical trials (VERTICAL/VISTA, and VELOCITY) comparing somavaratan vs daily rhGH for pediatric GH deficiency

Backeljauw Philippe , Miller Bradley S , Wright Nancy , Maniatis Aristides , Stalvey Michael , Humphriss Eric , Charlton R William , Bright George M

Efficacy of rhGH for treatment of pediatric GH deficiency (PGHD) may depend on several variables at treatment initiation (age, body weight, height velocity (HV), IGF-I SDS, skeletal maturation, GHmax), but may be compromised by poor adherence to required daily injections. Somavaratan is a novel, long-acting rhGH fusion protein that demonstrated clinically meaningful improvements in HV and IGF-I concentration in PGHD patients. Over 200 pre-pubertal GHD subjects have enrolled in...