I think the 202 study is doing what is it designed to. It is giving the team real time access to data that the blinded trial can't do. I don't think it was ever supposed to be statistically robust. That's the RCT format of the 201.
it would be interesting to know if these responders were in the biomuse study and whether they showed progression until the last 6 months when the disease seems to have stabilised. If they get that trend in say 15-20 201 early patients, then we are talking.
as for whether MSA is a big enough disease to be economically viable, I estimate about 9000 Americans with MSA. Neuren with Rett has about 5000. The Rett drug daybue is having a $400-500M first year in market. Imo, MSA is more than enough to be a compelling economical model.
the SP, that's another story. The market sale facility totally took the wind out of the sails of the report. Looks like a greedy cash grab, but maybe they got a $500k bill to allow then to get a licence to use the MRI volume AI...
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- Good to look at the reference study by Wenning et al
Good to look at the reference study by Wenning et al, page-5
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