Enrollment is open for a trial of AT2220, a drug designed to enhance the effects of enzyme replacement therapy in people with acid maltase deficiency
How a father led a quest to discover a treatment for his critically ill children, and ended up the subject of a major motion picture
John Crowley talks about what's needed in the "golden age" of drug development
The approval of Myozyme in 2006, after decades of research, has transformed Pompe disease from a severely disabling or fatal condition into a treatable, chronic disorder. But responses to the drug vary, its cost is high, and some questions remain unanswered.