Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

These are known as disease-modifying treatments and have been licensed for private use. Other medications are under review to see if they could be prescribed to people in the very early stages of ...