Gene Therapy May Be “One Shot Stop” for Rare Bone Disease | Newswise
Newswise — 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. “It’s been a tremendous success and has proven to be a lifesaving treatment,” said José Luis Millán, PhD, professor in the Human Genetics Program at…