IMPE Abstracts

To describe the BMD at FH of well-controlled PKU patients we retrieved retrospectively, data from 18 compliant moderate/severe patients (7 female), under conventional treatment since diagnosis. Eleven had received sapropterin dihydrochloride (BH4) since puberty, increasing their phenylalanine intake. FH, mid-parental height (MPH), genotype, fractures, and tolerance to phenylalanine intake (tol) before and after BH4 were considered. Total Body (TB) and Lum...

Introduction: X-linked hypophosphatemia (XLH) is a rare hereditary condition of phosphate wasting leading to rickets and short stature in childhood, additional adult manifestations, and lifelong stiffness, chronic pain, muscle weakness, and fatigue.Methods: UX023-CL401 (NCT03651505) is a prospective, multinational, multicenter, longitudinal, disease monitoring program (DMP) that aims to characterize the clinical, radiogr...

Setrusumab is a fully human anti-sclerostin monoclonal antibody in development for the treatment of osteogenesis imperfecta (OI). In a Phase 2b study in adults with OI, setrusumab demonstrated robust increases in bone formation, density and strength across OI Types I, III, and IV (NCT03118570). The Orbit study is an operationally seamless Phase 2/3 clinical trial assessing the efficacy and safety of setrusumab in pediatric and young adult participants with OI (NCT0512...