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Based on its mechanism of action, do you think sodium phenylbutyrate and taurursodiol could have benefit in other neurodegenerative disorders?

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Based on its mechanism of action, do you expect that sodium phenylbutyrate and taurursodiol will have benefit in other neurodegenerative disorders?

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3 Answers
Mednet Member
Mednet Member
Neurology · Duke
Answered on

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 presentation o...

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Neurology · University of Utah
Answered on

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...

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Mednet Member
Mednet Member
Neurology
Answered on

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...

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