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Preserving neurons. Protecting moments.

Our science is our humanity. From the individual neuron to the entire neurodegenerative disease community, we strive to enhance our knowledge, identify research opportunities, and grow our relationships to improve the lives of people with neurodegenerative diseases and of their families.

Amylyx’s mission to one day end the suffering caused by neurodegenerative diseases began with a simple question: “Why do neurons die?” Unlike most other cells in the body that regularly die and are replaced as part of healthy function, mature neurons are normally resistant to cell death.1 However, neuronal death can be triggered when multiple stress factors are activated, a circumstance commonly seen in neurodegenerative disorders.2

Most neurodegenerative disorders have complex pathophysiology, with multiple pathways contributing to eventually cause neuronal death. Our focus is on understanding and targeting these drivers of neuronal death with the goal of preserving not only neurons, but more moments that matter.

See how endoplasmic reticulum and mitochondrial dysfunctions contribute to ALS pathophysiology.

Mechanism of Disease
  1. Milo R, Phillips R. Rates and durations. In: Cell Biology by the Numbers. 1st ed. New York, NY: Garland Science; 2016:209-281.
  2. Chi H, et al. Int J Mol Sci. 2018;19(10):3082.
  3. Hardiman O, Al-Chalabi A, Chio A, et al. Amyotrophic lateral sclerosis. Nat Rev Dis Primers. 2017;3:17071.doi:10.1038/nrdp.2017.71.
  4. Kiernan MC, et al. Nat Rev Neurol. 2021;17(2):104-118.
  5. Fricker M, Tolkovsky AM, Borutaite V, Coleman M, Brown GC. Neuronal cell death. Physiol Rev. 2018;98(2):813-880. doi:10.1152/physrev.00011.2017.
  6. Manfredi G, Kawamata H. Neurobiol Dis. 2016;90:35-42.
  7. Smith EF, et al. Neurosci Lett. 2019;710:132933. doi: 10.1016/j.neulet.2017.06.052.
  8. Brown RH, Al-Chalabi  A. N Engl J Med. 2017;377(2):162-172. 2.
  9. Al-Chalabi A, et al. Lancet Neurol. 2016;15(11):1182-1194.
  10. Walker AK, et al. IUBMB Life. 2011;63(9):754-763.

Advancing Therapeutic Potential

Learn more about Amylyx’s investigative therapy, AMX0035 (sodium phenylbutyrate and taurursodiol).