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Nouvelles thérapies pour les maladies osseuses de l'enfant

Ballhausen, Diana
Dépraz, Nuria Garcia
Unger, Sheila
Bonafé, Luisa
Published in Revue médicale suisse. 2012, vol. 8, no. 329, p. 398-402
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.
Keywords Bone Diseases/drug therapyChildHumans
PMID: 22432239
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Other version: http://rms.medhyg.ch/numero-329-page-398.htm
Research group Gastroentérologie et transplantation (pédiatrie) (181)
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BALLHAUSEN, Diana et al. Nouvelles thérapies pour les maladies osseuses de l'enfant. In: Revue médicale suisse, 2012, vol. 8, n° 329, p. 398-402. https://archive-ouverte.unige.ch/unige:31213

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Deposited on : 2013-11-20

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