quoting from SA article:
"As with any clinical trial, sample size is extremely important to demonstrate reproducibility and to identify both efficacy/side-effects. A small trial may not provide an accurate summary of a drug profile and a small trial may not have the power to capture the genetic variation that may actually occur in the real-world patient population. It is important to note that patient germline genetic variation can impact the efficacy/toxicity of a drug response"
Genetic variation ??? Patients have a common genetic variation! That's why they lack dystrophin and that's why they've been selected. There's only one genetic variation that matters here: Exon 51!! and it's been dealt with -to a seemingly significant extent- by the drug.
Regarding 6MWT (clinical benefit) , the author muddles things up and concludes that results are unreliable. We know one thing for sure though: without Etiplirsen, those same boys would defiinitively be worse off.
Finally, about side effects. of all antisense chemistries, PMO is the safer.