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recAP improves mineralization in hypophosphatasia models
January 2015
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BUNNIK, The Netherlands—AM-Pharma B.V. recently shared preclinical data on its drug candidate recAP (recombinant human Alkaline Phosphatase) for the treatment of the rare disease hypophosphatasia, which appeared in Bone. Hypophosphatasia, a mineralization disorder that causes soft bones (rickets or osteomalacia) and defects in teeth and periodontal tissues, affects an estimated one in 100,000 newborns. AM-Pharma's paper detailed how recAP increased mineralization of bone and teeth and prevented seizures. Mice with severe hypophosphatasia received daily subcutaneous injections of recAP in 1, 8 or 16 mg/kg doses from birth up to 53 days, and as a result saw normal lifespans and body weights, while untreated mice died after 20 days. Imaging showed improved mineralization of cortical and trabecular bone as well as secondary ossification centers in longer bones. Additionally, treated mice displayed no evidence of craniosynostosis or ectopic calcification.
 
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