Where's the Sarepta data we were promised?

Back in 2016, when Sarepta Therapeutics won a controversial approval for a rare disease drug, it promised to run a study confirming the treatment’s benefits. Three years later, that trial is yet to treat a single patient.

As STAT’s Adam Feuerstein reports, Sarepta said it’s working as fast as possible. In the meantime, however, the company is reaping about $300 million a year from a Duchenne muscular dystrophy treatment whose clinical benefit has never been demonstrated.

One doctor who treats patients with the condition told STAT that some families have reported their children have received a benefit from Exondys 51; other families have not and have discontinued treatment.

Read more.