This approval is another example of the benefits of the Orphan Drug program, which provides incentives for the development of treatments for diseases affecting fewer than 200,000 patients in the United States a year. Until now, Pompe disease has had no approved treatment.  -   Dr. Galson     Quotes
This approval is another example of the benefits of the Orphan Drug program, which provides incentives for the development of treatments for diseases affecting fewer than 200,000 patients in the United States a year. Until now, Pompe disease has had no approved treatment. Dr. Galson
 Approval