Essentially the efficacy test is a 3 point scale that measures standing and walking. It's not granular enough to get real statistical significance on, and never will be unless another kind of test is defined. So the tests are basically always doomed to fail rigid scrutiny by the FDA.
In the meantime they're getting dramatically effective results, and Peter Marks is bucking the FDA's traditionally conservative stance to make the product available to the few who desperately need it. I'll eat my words if someone finds he was paid off for this. I think is a truly miraculous kind of therapy, and I hope to one day have regularly available gene therapy for my own afflictions.
https://www.reddit.com/r/technology/comments/1dlsset/comment...