• Nov 17, 2015 | Webinar

    Hypophosphatasia: The Journey to Treatment

    Michael P. Whyte, M.D.

    Supported by an educational donation provided by Alexion Pharmaceuticals

    Alkaline phosphatase (ALP) is the most frequently assayed enzyme, and elevated serum ALP is concerning for skeletal disease. I will briefly review how the function of ALP became largely known from the discovery and then study of the remarkably informative inborn-error-of-metabolism called hypophosphatasia (HPP). HPP is the heritable rickets or osteomalacia that features low serum ALP activity, spans the greatest range of severity for any skeletal disease, and represents the last rickets or osteomalacia to have a medical treatment. The insights concerning ALP from HPP became fundamental to treating this disorder, now focused largely on bone-targeted, ALP-replacement (asfotase alfa) recently approved in Japan, Canada, Europe, and the United States.

    As a result of the webinar participants will be able to:

    • Discuss HPP’s remarkably broad-ranging severity.
    • Identify (diagnose) HPP in pediatric and adult patients.
    • Review supportive and enzyme-replacement treatment for HPP.

    Length: 60 minutes

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