My last (longer) post on $SRPT after today's info:
10/10 kids stable! I just can't believe it. The stock should be up 10% tomorrow. Irrefutable evidence the drug works. This is what we have:
100% of the kids produced dystrophin. We know the levels of dystrophin are what you'd expect to need to replicate Becker's.
100% of the kids (minus the twins) are stable once the drug has kicked in. Wow! No outliers. Everyone thought it was 1 or 2 or 3 kids that were thriving. Nope; all 10 are stable. Simple amazing.
Anecdotal evidence from the parents say their boys are performing many functions they couldn't do before the trial. This is really the best evidence the drug works, even if it can't be used as data in a clinical study.
Safety has been great over 1.5 years. The FDA said they will require a few months of P3/confirmatory safety before granting AA. This is to cover their #$%$.
If you're the FDA, how do you deny this AA? You have 100% of the boys benefiting clinically w/ 100% of them having a biomarker as evidence. The twins lost almost all functionality before the drug entered their body. Chris said 8 weeks into the trial they were below 250 meters. We know at 12 weeks the drug hadn't worked as the 50MG boys showed no dystrophin at that timeframe. This is not data mining.
Chris did a fantastic job today. The transparency with the individual data should be enough to shut up the critics. What do you have left? It's all placebo effect? Single center study? All manipulated? Boys just "wished" they could produce dystrophin?
Really am excited at todays data. Kudos to the company for releasing it. They didn't have to - only the FDA needs to see it - but I think it will silence critics.