Duchenne muscular dystrophy – Elaine Schattner

Elaine Schattner

writer, health care advocate, physician

The FDA Did The Right Thing By Approving a Novel Drug For A Disabling Disease

The FDA’s decision reflects a thoughtful, not knee-jerk approach to its mission, which is to protect the public from fraudulent and unsafe substances. What’s at issue is how well eteplirsen works. And that it’s too expensive.

Lip Service To Patients And Caregivers, Or Respect? The Significance Of An FDA Panel’s Review

..as if mothers telling an FDA panel about what’s happening to their kids with Duchenne muscular dystrophy, a disabling condition, is the opposite of science.