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AMX0035 is a drug designed to prevent nerve cell death and degeneration. The drug improved cellular metabolism and promoted protein folding in cellular and animal models of neurodegenerative disease. When metabolism and protein folding are not functioning properly, neurons halt key functions and ultimately can die. Metabolism and protein folding appear to be dysfunctional in ALS, Alzheimer’s and other neurological diseases. AMX0035 helps to boost metabolism and synthesize proteins called heat shock proteins that help to fold other proteins into their proper shapes. Through these mechanisms AMX0035 prevents neuronal death and degeneration. AMX0035 is currently under evaluation to see if it can demonstrate these beneficial effects in patients. If neurons are able to stay functional, it may delay loss of daily functioning in ALS, Alzheimer’s disease and other diseases caused by nerve cell death.

Amylyx Pharmaceuticals was founded in 2013 with the mission of improving the lives of patients with neurodegenerative diseases. The company’s lead therapeutic, AMX0035, is designed to simultaneously target pathways originating in the mitochondria and endoplasmic reticulum which lead to neuronal death and degradation. AMX0035 is currently in clinical trials in both Alzheimer’s disease and ALS.

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1 floor Amylyx Pharmaceuticals [*.*.*.*] published in 2020/07/02/ 08:33:00
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Amylyx Pharmaceuticals was founded in 2013 with the mission of improving the lives of patients with neurodegenerative diseases.

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