The approval comes amid a flurry of research showing broader promise for the approach in a range of disorders, from a rare form of blindness to hemophilia to heart failure.
Aside from the hemophilia drugs, the company is also expecting data from a drug for amyotrophic lateral sclerosis and there could be an expert panel for its BG-12 multiple sclerosis pill by the end of the year, with approval expected early in 2013.
For these kids, suffering from diseases such as hemophilia, sickle cell anemia and cancer, it is a chance to see past the walls of the hospital to what the world might be like if a diagnosis didn't control their every move, if clinics and hospital rooms weren't like second homes.