title: [New therapies for children affected by bone diseases].
abstract: Considerable progress has been achieved in recent years in treating children affected by bone diseases. Advances in the understanding of the molecular pathophysiology of genetic bone diseases have led to the development of enzyme replacement therapies for various lysosomal storage diseases, following the breakthrough initiated in treating Gaucher disease. Clinical studies are underway with tailored molecules correcting bone fragility and alleviating chronic bone pain and other manifestations of hypophosphatasia, or promoting growth of long bones in achondroplasia patients. We further report our very encouraging experience with intravenous bisphosphonate treatment in children suffering from secondary osteopenia and the high prevalence of calcium and vitamin D deficits in these severely disabled children.
authors.affiliation: Division de pédiatrie moléculaire, CHUV et Université de Lausanne, 1011 Lausanne. diana.ballhausen@chuv.ch
authors.forename: Diana
authors.initials: D
authors.lastname: Ballhausen
authors.forename: Nuria Garcia
authors.initials: NG
authors.lastname: Dépraz
authors.forename: Ilse
authors.initials: I
authors.lastname: Kern
authors.forename: Sheila
authors.initials: S
authors.lastname: Unger
authors.forename: Luisa
authors.initials: L
authors.lastname: Bonafé
mesh_terms: D001847:Bone Diseases / Q000188:drug therapy*; D002648:Child; D006801:Humans