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). This FDA-approved therapy is based on a Sanford Burnham Prebys scientist’s decades of research on the TNAP enzyme and his laboratory’s studies demonstrating preclinical safety and efficacy.

In a paper published January 12, 2025, in the Journal of Bone and Mineral Research, researchers added additional weight to prior preclinical evidence of the safety and effectiveness of a gene therapy for HPP. 

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