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Sodium Phenylbutyrate-Taurursodiol Promising for ALS

Slower functional decline seen with active drug versus placebo through 24 weeks

THURSDAY, Sept. 3, 2020 (HealthDay News) — For patients with amyotrophic lateral sclerosis (ALS), function declines more slowly through 24 weeks in those receiving sodium phenylbutyrate-taurursodiol versus placebo, according to a study published in the Sept. 3 issue of the New England Journal of Medicine.

Sabrina Paganoni, M.D., Ph.D., from Massachusetts General Hospital in Boston, and colleagues enrolled participants with definite ALS who had had an onset of symptoms within the previous 18 months. Participants were randomly assigned to receive either sodium phenylbutyrate-taurursodiol or placebo in a 2:1 ratio (89 and 48 participants, respectively).

The researchers found that the mean rate of change on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised score was −1.24 versus −1.66 points per month with the active drug versus placebo through 24 weeks (difference, 0.42 points per month) in the modified intention-to-treat analysis. There was no significant difference between the groups in secondary outcomes. With the active drug, adverse events were mainly gastrointestinal.

“Longer and larger trials are necessary to evaluate the efficacy and safety of sodium phenylbutyrate-taurursodiol in persons with ALS,” the authors write.

The study was partially funded by Amylyx Pharmaceuticals.

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