Amyotrophic Lateral Sclerosis (ALS)

Rapidly progressing neurodegenerative disease caused by oxidative damage which leads to neuronal cell death and muscular atrophy

What is Amyotrophic Lateral Sclerosis?

Amyotrophic lateral sclerosis, sometimes referred to as ALS, motor neuron disease, or Lou Gehrig’s disease, is a rare, progressive, and fatal neurodegenerative disease that affects motor neurons in the brain and spinal cord.^1,2Amyotrophic lateral sclerosis becomes life-threatening as people with this disease ultimately lose the ability to move, speak, eat, and breathe.¹

How common is Amyotrophic Lateral Sclerosis?

Approximately 1.5 to 3 per 100,000 people develop amyotrophic lateral sclerosis every year in North America and Europe, which equates to approximately 5,000-6,000 new cases each year.^1,4,5 Men are disproportionately affected at a ratio of 2:1.^4,5

How is PTC Working to Treat Amyotrophic Lateral Sclerosis?

Utreloxastat, discovered through PTC’s Ferroptosis & Inflammation platform, is an investigational small molecule that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress, protein aggregation and inflammation response pathways that underpin neurodegenerative disease pathology.

The possibility to do something that improves the lives of patients is always motivating, particularly for a disease as deadly and devastating as ALS.

Marla Weetall SVP, Pharmacology & Biomarkers, PTC

Do you have questions?

Please reach out if you would like to speak with us.

Patients and Families Healthcare Professionals

References

[1] Kiernan MC, et al. Amyotrophic lateral sclerosis. The Lancet, 2011; 377(9769):942-955.

[2] Dhasmana S, et al. The panoramic view of amyotrophic lateral sclerosis: A fatal intricate neurological disorder, Life Sciences 2022;288:120156. https://doi.org/10.1016/j.lfs.2021.120156.

[3] Kiernan MC, et al. Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nat Rev Neurol 2021;17:104–118.

[4] Hulisz, Am J Manag Care. 2018;24:S320-S326,

[5] Vucic et al. Int J Mol Sci. 2019;20(11):2818;

[6] Morrison et al., Expert Rev Mol Med, 2006;8(11);

[7] Chio et al., Amyotroph Lateral SCler/. 2009; 10(5-6)