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KRN23 therapy improves rickets scores
August 2015
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NOVATO, Calif.—July saw Ultragenyx Pharmaceutical Inc. report positive interim data from the first 12 patients in its ongoing pediatric Phase 2 study of KRN23, its recombinant human monoclonal antibody against fibroblast growth factor 23 (FGF23) for the treatment of X-linked hypophosphatemia (XLH). The study is being conducted under a collaboration and license agreement with Kyowa Hakko Kirin to develop and commercialize KRN23. Five of the patients in the biweekly dosing group saw an 80-percent reduction in rickets score, while three patients in the monthly dosing group saw a 38-percent reduction in rickets score (two saw no change and one worsened by 0.5 points). No serious adverse events have been reported to date, nor have there been any study discontinuations. The most common adverse events thought to be treatment-related were injection site reactions.
 
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