Families of boys suffering from Duchenne muscular dystrophy push for approval of a new medicine because there are no other treatments. But scientists point out that there is no evidence that the drug works. Does it set a dangerous precedent for FDA to approve a new drug based on patients’ claims rather than science?
NCHR President, Diana Zuckerman, and NCHR Senior Fellow, Laura Gottschalk, are quoted in this article explaining their concerns about the possible approval of a drug with no solid evidence proving it works.
Read the full article here.