ISSN 1662-4009 (online)

ey0021.5-12 | Novel Treatments | ESPEYB21

5.12. Once-weekly transCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-Blind, placebo-controlled, dose-escalation trial

Savarirayan Ravi , Hoernschemeyer Daniel G. , Ljungberg Merete , Zarate Yuri A. , Bacino Carlos A. , Bober Michael B. , Legare Janet M. , et al.

In brief: This multinational, randomised, double-blind, placebo-controlled, dose-escalation Phase 2 study evaluated the safety and efficacy of TransCon CNP (navepegritide), a C-type natriuretic peptide (CNP) analogue designed to allow continuous CNP exposure with once-weekly dosing, in 42 children with achondroplasia aged 2-10 years. The study was sponsored by Ascendis Pharma.Commentary: Achondroplasia is one of the most common constitutional bone disord...

ey0021.5-5 | Advances in Clinical Practice | ESPEYB21

5.5. The IMPACT survey: a mixed methods study to understand the experience of children, adolescents and adults with osteogenesis imperfecta and their caregivers

Ingunn Westerheim, , Hart Tracy , van Welzenis Taco , Lande Wekre Lena , Semler Oliver , Raggio Cathleen , Bober Michael B. , Rapoport Maria , Prince Samantha , Rauch Frank

In brief: The IMPACT Survey collected a comprehensive dataset (including demographics, clinical characteristics and clinical signs, symptoms and events and their impact) on the experience of individuals with osteogenesis imperfecta (OI) (n=2312 individuals across self- and proxy reports), and their caregivers (n=560). Individuals with OI reported numerous and evolving symptoms that affect their quality of life, notably pain and fatigue, which are consistently present.<p cl...