Based on its mechanism of action, do you think sodium phenylbutyrate and taurursodiol could have benefit in other neurodegenerative disorders?
Answer from: at Community Practice
Yes. Mitochondrial dysfunction (Kim et al., PMID 35682574) are present and believed to be important in driving the progression of several neurodegenerative diseases. Amylyx is currently investigating this drug in patients with Alzheimer's disease (Amylyx Pharmaceuticals announces oral presentat...
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Answer from: at Academic Institution
This is difficult to answer as the proposed mechanisms of action of the two drugs are minimally described in the article by Paganoni et al, PMID 32877582, listing reduction of endoplasmic reticulum stress and mitochondrial dysfunction for sodium phenylbutyrate and reducing reduce cell death for taur...
Answer from: at Community Practice
Yes, in fact, this drug combination is also being evaluated for other neurodegenerative diseases including Alzheimer's disease (AD). The PEGASUS trial was a biomarker-driven phase 2 trial in AD; in the trial, treatment with AMX0035 resulted in significant changes in several biomarkers that have rele...