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CNM-Au8 in Amyotrophic Lateral Sclerosis—Reply

Summary by JAMA Network
In Reply We appreciate the letter from Drs Endo and Kami about the HEALEY ALS Platform Trial, a randomized, placebo-controlled trial of CNM-Au8 for ALS. They raise a complex question with 3 parts: (1) was there a failure of randomization in this trial; (2) could the statistically significant imbalance in race and ethnicity have affected the outcome of the trial; and (3) why was the percentage of White participants in a US-based trial so high?

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JAMA Network broke the news in on Tuesday, July 15, 2025.
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