By Mindy LefflerAug. 20, 2026
Leffler developed the Duchenne Video Assessment, used in Capricor’s phase 3 study, and consults on its data.
Five years ago, we moved my son into a college dorm. I know that makes us sound like thousands of other families with 18-year-olds, but my son has Duchenne muscular dystrophy. He couldn’t walk, and because of his waning arm strength, we all thought his college independence would be short-lived.
Instead, we are now getting ready to move him into that same dorm again, where he still lives independently, so he can begin the second year of his master’s degree study. He has spent those five years on Capricor’s deramiocel, a drug that received a negative FDA advisory committee vote last week on its secondary outcome: the definitiveness of its ability to stabilize heart function in a population of boys that included ones whose hearts were still stable. It was much noisier data than the significance seen in the subpopulation that already had signs of heart dysfunction.
The decision demonstrates something important: Designing a rare disease study consists of hundreds of tiny decisions. Sponsors make them with the best knowledge they have at the time but with no way of predicting each decision’s impact with high degree of certainty because they are the ones blazing the trail. But lots of sick people’s lives depend on getting these little decisions right.
