What to hopefully look forward too, page-29

Hi Gaz. I don't recall any disagreement with pivalde. IMO he is the best poster on this site.
Regarding BIIB's Aducanumab, that is a good illustration of what I was saying about patient numbers and safety requirements. The aducanumab trials enrolled thousands of patients. Biogen is a powerful company capable of funding huge trials. For a little Bio company with a treatment for an orphan disease unmet need, obviously the FDA will have to make do with much smaller data sets, and they do that. What they don't usually compromise on is safety. That means enough patients treated to meet FDA safety standards.
Accelerated approval does not mean instant or very near approval. Your Vyondys example was given a multi year trial to complete. 2019 to 2024.
With rare, life threatening diseases, and no existing approved treatment, a conditional approval hopefully is possible
All just my opinion as an investor..