"However, the decision was made against the recommendation of...

"However, the decision was made against the recommendation of several agency staff members, who wrote that there was "significant uncertainty regarding the benefits of the treatment."

"Last October, Sarepta released the results of a confirmatory trial showing that the treatment missed its primary goal, which was a measure of how well patients could move. However, company executives said that the trial was able to meet secondary and exploratory endpoints, which they argued showed that the treatment's mechanism of action worked and could slow the progression of the disease."

"Despite the review team's conclusion, Marks said that "the available data are compelling, including the positive benefit shown on the secondary endpoints and the exploratory evidence" from clinical trials. He also noted that there is a lack of alternative treatments for Duchenne muscular dystrophy.

Two of Marks' top lieutenants also objected to Marks' decision. In a letter to Marks, Lola Fashoyin-Aje, director of the Office of Clinical Evaluation, and Nicole Verdun, super office director of the Office of Therapeutic Products, said that Elevidys failed two consecutive, placebo-controlled trials in four- to seven-year-olds."

Significant difference in the two situations. You see Marks as the problem - that is clearly your biased opinion.

It is interesting that the trials were placebo-controlled for kids 4-7. Maybe MSB has played a part in future FDA trial designs for children?