Where are the exceptional responders?
Datamethods Discussion Forum [Unofficial]
May 12, 2026
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:
- 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