hmmm a lot of half empties around here...
At the moment, I'm in a bit of a wait and see mode (especially given I bought some more just before the Epichem liquidation announcement (aggh)). Anyways, I'm actually quietly confident about Sam's emails' confidence but I want to see first hand why he's seemingly coming across so confident. Undercanstumble?
First off about the neurofilament light chain (NfL) biomarker: as @Quiltman already demonstrated, this is just a favoured marker but not rolled gold guaranteed. Put it another way, if the health outcomes of the patients, like, say, stability over 9 months and the other 2 biomarkers already announced are contradicted by the NfL findings, then I'll discount NfL first and everyday. If you want to understand the scientific reason for this, here it is: (thanks Quilts): https://academic.oup.com/brain/article/146/7/2711/6780887
Second, when dealing with projects like this, before starting to spout off cyncism and negativity, ask yourself why and how we got here. And the reason is very simple: BECAUSE THE PRINCIPAL INVESTIGATOR OF THIS TRIAL, ONE Dr Mathers, WANTED THIS INTERIM TRIAL. And why did she do that? TO "EXPEDITE" THIS PHASE ONE TRIAL IN DOUBLE QUICK TIME SO AS TO START THE NEXT TRIAL ASA FFFFING P !!!!!
AND WHY WOULD A PROFESSOR AND MEDICAL RESEARCHER OF MONASH UNIVERSITY AND PRACTICING MND/ALS CLINICIAN ASK FOR SHORT CUTTING THE TRIAL TO THE NEXT STAGE????
Anybody got a clue???
Oh, and wait, TGA rules are not identical to FDA rules on orphan drug status. Look them up. Just saying.
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