Skeletal Health and Dysplasia
PROGRAM OVERVIEW
Our Skeletal Health and Dysplasia team focuses on understanding bone related genetic disorders in children. Our research is currently involved in multiple multicenter studies investigating pediatric achondroplasia, mucopolysaccharidoses and osteogenesis imperfecta treatment across the world.
RESEARCH HIGHLIGHTS
This year, we launched the Propel OLE and Tyra( Beach 301) clinical trials investigating new oral treatments for achondroplasia, while continuing patient participation in the Attach, Propel and Propel 3 trials for achondroplasia, the ACCEL series for hypochondroplasia and the Cosmic trial for infusion therapy in osteogenesis imperfecta( OI). In parallel, the Achondroplasia Biobank was established to collect biological samples and health data from patients and family members to study variations in outcomes and inform prevention and treatment strategies. Beyond clinical trials, our team works with the Skeletal Dysplasia Multidisciplinary Care Clinic to explore and describe unusual diagnoses and optimize care for patients with these rare disorders.
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