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Where are the exceptional responders?

Datamethods Discussion Forum [Unofficial] May 12, 2026
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It’s fascinating to contrast how this NYT piece (rightly, IMO) extols the exceptional responses from a trial of tofersen in SOD1 -ALS, whereas the formal study report [1] squirrels these extraordinary findings away in the Results, Discussion and Supplement 1!

The abstract gives no hint of the exceptional responses:

Here is how the Discussion couched the obvious in statistical language:

From Supplement 1:

  1. Miller TM, Cudkowicz ME, Shaw PJ, et al. Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis. JAMA Neurol. 2026;83(2):115. doi:10.1001/jamaneurol.2025.4946

Discussion in the ATmosphere

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