Novel Therapeutic Agents for Rare Diseases of Calcium and Phosphate Metabolism
Horm Metab Res DOI: 10.1055/a-1917-0519The last decade has been revolutionary regarding the management of rare bone
diseases caused by impaired calcium and phosphate metabolism. Elucidation of the
underlying genetic basis and pathophysiologic alterations has been the
determinant factor for the development of new, disease-specific treatment
agents. The phosphaturic hormone Fibroblast Growth Factor 23 (FGF23) possesses a
critical role in the pathogenesis of various hypophosphatemic disorders. Among
them, the genetic disorder of X-linked hypophosphatemia and the acquired
syndrome of tumor-induced osteomalacia, although very rare, have attracted the
scientific community’s attention towards designing an FGF23-inhibitor as
a potential specific therapy. The monoclonal antibody burosumab was approved for
the treatment of children and adult patients with X-linked hypophosphatemia and
recently for tumor-induced osteomalacia patients, demonstrating benefits
regarding their symptoms, biochemical profile and bone mineralization status.
Asfotase alfa is a hydroxyapatite-targeted recombinant alkaline phosphatase, an
enzymatic replacement therapy, substituting the defective activity of tissue
non-specific alkaline phosphatase, in patients suffering from hypophosphatasia.
Promising data regarding its favorable effect on survival rate...
Source: Hormone and Metabolic Research - Category: Endocrinology Authors: Roumpou, Afroditi Yavropoulou, Maria P Chronopoulos, Efstathios Kassi, Eva Tags: Review Source Type: research
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