Abstract
Fibrodysplasia ossificans progressiva (FOP) is a rare heritable disorder characterized by the progressive accumulation of heterotopic bone arising in the connective tissues of skeletal muscle, ligaments, and tendons. Although the mechanism underlying the formation of heterotopic bone in FOP is at present undefined, the formation of cartilage and bone in soft tissue is preceded by the appearance of proliferating fibroblastic-like cells, presumed to represent either chondroblast or osteoblast precursors. Since retinoic acid and several synthetic analogs have been shown to exert potent inhibition over the differentiation of cartilage precursor cells both in tissue culture and in the mammalian fetus, these drugs would appear to represent a rational therapeutic modality in FOP. We wish to report our initial findings of the effect of Ro 10-9359, a synthetic retinoic acid derivative, on heterotopic bone formation in two children with FOP treated over a period of 6 months. Although these patients have received Ro 10-9359 for too short a period to permit a conclusive assessment of efficacy the initial clinical response appears sufficiently favorable to warrant continued investigation in these and other patients.
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Zasloff, M., Schulman, J. 779 TREATMENT OF FIBRODYSPLASIA OSSIFICANS PROGRESSIVA WITH THE SYNTHETIC RETINOID, Ro 10–9359. Pediatr Res 15 (Suppl 4), 572 (1981). https://doi.org/10.1203/00006450-198104001-00803
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DOI: https://doi.org/10.1203/00006450-198104001-00803