Gene therapy may be ‘one shot stop’ for rare bone disease

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week. Currently, patients are treated with injections of asfotase alfa, a mineral-targeted form of the missing enzyme called tissue-nonspecific alkaline phosphatase (TNAP).

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