Gene Therapy May Be 鈥淥ne Shot Stop鈥 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,...
3-Feb-2025 9:10 PM EST
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