"Notably, they rejected Sarepta’s arguments that Elevidys’...

"Notably, they rejected Sarepta’s arguments that Elevidys’ benefits on secondary measures in its main trial make up for the fact its primary outcome was negative. Because that primary statistical test failed, the secondary findings are “misleading” and “cannot guide any stakeholders — including patients, family members and caregivers, and prescribers — in making informed decisions” about Elevidys’ potential benefits, wrote clinical reviewers Mike Singer and Xiaofei Wang.

“We cannot reliably distinguish if these results are due to actual effects of Elevidys, or to chance alone,” they wrote.

Marks took an opposite view, finding Sarepta’s secondary study findings “compelling.” He also argued that historical data suggests even a small benefit compared to a placebo in someone with Duchenne is “clinically meaningful.”"

Hope he takes this view with the DREAM-HF trial...