IMPE Abstracts

Osteogenesis imperfecta (OI) is a hereditary skeletal disease characterized by bone fragility. Bone mineral density (BMD), evaluated by dual-energy X-ray absorptiometry (DXA), is used to assess bone brittleness. The height-for-age Z-score (HAZ)-adjusted BMD-for-age Z-score (BMDHAZ) to reduce the confounding factor of short stature is calculated in children and adolescents with OI. However, even with BMDHAZ, severity evaluation in children and adolescents ...

Hypophosphatasia (HPP) is a congenital skeletal disorder characterized by failure of bone calcification, which is caused by deficiency of the tissue non-specific alkaline phosphatase (TNSALP) enzyme. There is a remarkable range of severity in the phenotype, and some pediatric HPP patients show failure to thrive depending on their severity. In this study, we aim to elucidate the association between serum ALP activity and growth in HPP children. Twenty-six HPP patients (16 males...

Background: Vosoritide increases annualized growth velocity (AGV) in children with achondroplasia aged 5 to 18 years. This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.Methods: This study compared once-daily subcutaneous administration of vosoritide, at doses of 15 or 30 mg/kg of b...

Objectives: Vosoritide is a potent stimulator of endochondral bone growth and is in development for the treatment of achondroplasia, the most common form of disproportionate short stature. We previously reported on a 52-week, phase 3, pivotal study that demonstrated a significant improvement in annualized growth velocity (AGV) when vosoritide was compared to placebo in children with achondroplasia aged 5-18 years (Savarirayan et al, Lancet, 2020). Thi...