Great find shyaam. Appreciate the information and analysis. It would be nicer if the results from the small P2a cohort were less dispersed. Three out of the nine patients (33%) fell outside the 95% confidence limits of -1.33 to 3.11.
Just for reference to others interested and to save them diiging up the results, the PUL 2.0 scores in the P2a - 24 week - study were:
I think the positive outcome from the trial was that 7 out of the 9 patients had no deterioration in their strength, and four out of the 9 actually increased, which was unheard of, I think, until this trial.
Anyway, the disparity in results and the small sample make the larger trial a little riskier than I would like (a shoo in would have been nice), given your findings on the likely placebo mean for 6 months of -1.09 (with steroid treatment or without?).
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