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author：focus 2025-10-02 06:18:55 - browse：88

Adam's take main illustration — Molly Ferguson/STAT

This week, a preview of the TUDCA study in ALS and thoughts on how it might impact Amylyx Pharmaceuticals. Also, I chatted with CRISPR Therapeutics CEO Sam Kulkarni about its new investor. Read to the end for some burning questions, random thoughts.

The next ALS data readout — overdue but important

A European academic consortium is expected to announce results soon from a clinical trial investigating the use of a widely available nutritional supplement called tauroursodeoxycholic acid, or TUDCA, in people living with ALS. The outcome of the TUDCA-ALS study could have implications for Amylyx Pharmaceuticals, because TUDCA is one of the two main ingredients in Relyvrio, the company’s approved drug for ALS.

In a recent interview at Amylyx’s Kendall Square headquarters, co-CEOs Justin Klee and Josh Cohen mostly downplayed the suggestion that results from the randomized, placebo-controlled TUDCA study might affect Relyvrio negatively, or even positively.

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