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. "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 Sanford Burnham Prebys.
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 Sanford Burnham Prebys.
