Gene Therapy May Be "One Shot Stop" for Rare Bone Disease
February 04, 2025
February 04, 2025
LA JOLLA, California, Feb. 4 -- Sanford Burnham Prebys issued the following news release:
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Gene therapy may be "one shot stop" for rare bone disease
New study adds to evidence that a single shot may be effective against a skeletal disease that currently requires nearly daily injections to treat
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delive . . .
* * *
Gene therapy may be "one shot stop" for rare bone disease
New study adds to evidence that a single shot may be effective against a skeletal disease that currently requires nearly daily injections to treat
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delive . . .