Does Medical Activism Actually Work?

This question entered my mind when I read the compelling story, “Moms, Regulators, Biotech Startups, and the Battle Over a Potentially Life-Saving Drug,” in Bloomberg BusinessWeek. Although the article doesn’t provide an answer, it seems to hint that it does.

The focus of the feature is Duchenne muscular dystrophy, a rare genetic disease that primarily affects boys. It describes the struggle of several affected families who are seeking care for their sons, as well as the role of FDA and the efforts of biotech companies to get a drug into the market.

As you read through the story, it is easy to identify with the parents’ struggle. At times, you may feel thankful that it is not your story, but want to help in the family’s struggles. You also get insight into the role social media can play in how “scientific” decisions are influenced.

Why did the FDA seem to reverse course on eteplirsen but allow Sarepta Therapeutics to move ahead in seeking regulatory approval? What do you think?