Hi Pete,
Your investigation got me thinking about the mortality rates in other MND clinical trials and how mortality affects the endpoints.
The following article may be helpful for a deeper understanding of the issue.
In particular, Table 1 documents survival percentages in previous clinical trials.
Our trial of !2 patients is a very small sample size, but the fact they have all survived to date is very encouraging. And of course if their quality of life has been maintained or improved then Phase 2 should be very very interesting.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8940672/
cheers, Thrifty
FDA Application for Orphan Drug Designation , PAA, MPL, page-23
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