IMPE Abstracts

Objectives: Fibrous dysplasia (FD) is a rare disorder resulting in fractures, pain, and skeletal deformities. Abnormal osteoprogenitor cells produce excess FGF23, leading to hyperphosphaturia in most patients and frank hypophosphatemia in a smaller subset. Studies suggest that FGF23-mediated hypophosphatemia is associated with increased FD-related morbidity. However, the relationship between serum phosphorus levels and skeletal complications has not been inves...

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...

Background: Achondroplasia (ACH) is the most common short-limbed skeletal dysplasia, affecting between 1 in 15,000 to 1 in 30,000 live births. People with ACH are at risk for several significant co-morbidities, including foramen magnum stenosis, obstructive sleep apnea, chronic otitis media with conductive hearing loss, spinal stenosis, and a propensity towards obesity. PROPEL is a prospective, non-interventional study designed to examine baseline growth param...

Background: Achondroplasia (ACH) is the most common form of short-limbed skeletal dysplasias and is caused by an activating pathogenic variant of the fibroblast growth factor receptor 3 (FGFR3) gene. People with ACH are at risk for several significant co-morbidities, including foramen magnum stenosis, obstructive sleep apnea, chronic otitis media with conductive hearing loss, spinal stenosis, and a propensity towards obesity. Decreased bone mass was r...

Background: Bone age (BA) is commonly used in pediatrics to define skeletal maturity for medical and non-medical purposes. Normal range is represented by 2 standard deviations (SDs) &pm; the mean. A BA > &pm;2 SDs from the chronological age (CA) is considered abnormal. BA in achondroplasia (ACH) has not been fully characterized; calculation is challenging given difficulties in comparing x-rays with standard radiographs if using the Greulich-Pyle (G&P) m...