ba0004oc7 | (1) | ICCBH2015
Hoyer-Kuhn Heike
, Netzer Christian
, Hero Barbara
, Schoenau Eckhard
, Semler Oliver
Osteogenesis imperfecta is a rare disease leading to multiple fractures, skeletal deformities and scoliosis due to a reduced bone mass. Pathological fractures caused by inadequate traumata are the most severe symptom. More than 85% of patients are affected by mutations in COL1A1/A2 impairing quantity and quality of collagen. At present no approved drugs for OI treatment in childhood are available. A single centre prospective pilot study was performed to assess safety and effic...