Osteogenesis imperfecta: new treatment options.
Classic treatments of osteogenesis imperfecta for children and adults include rehabilitation therapy and orthopedic surgery. New treatments include bisphosphonates. Among them, the effects of intravenous pamidronate have been primarily evaluated in children in several open cohorts. Treatments of adult patients are not established. Several other treatments may have an effect on the fracture rate. However, further studies are needed to demonstrate an effect on fracture rate in children and adult patients. A new approach has been developed to replace mutated genes using mesenchymal stem cells and a construct, which inactivates COL1A1.
Chevrel
Guillaume
G
Department of Pediatrics, Edouard Herrriot Hospital, 5, place d'Arsonval, 69437 Lyon Cedex 03, France.
Cimaz
Rolando
R
D000328:Adult; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D004164:Diphosphonates / Q000627:therapeutic use*; D015316:Genetic Therapy; D006801:Humans; D007275:Injections, Intravenous; D010013:Osteogenesis Imperfecta / Q000188:drug therapy* / Q000235:genetics; D000077268:Pamidronate
D000893:Anti-Inflammatory Agents; D004164:Diphosphonates; D000077268:Pamidronate