They show slight increases in dystrophin, it’s just a case of...

They show slight increases in dystrophin, it’s just a case of where you draw the line and define “efficacy”. It’s thought that at least 10% of normal dystrophin levels are required for any noticeable improved physical effect, and the improvements caused by the drug still leave the child with less than 1% of normal levels.

I go back to a point I made over a year ago, in light of DMD parents signing a petition that influenced the FDA’s decision - what Sarepta are selling to parents of newly diagnosed DMD patients is “hope” at little expense to those who get full health coverage, and at a stage when the prognosis is horrible to contemplate, but not yet played out.

Parents would do anything to keep hope of a cure or just an improvement alive.

The EMA rejected the application.