Amyotrophic Lateral Sclerosis (ALS)
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.1,2 Amyotrophic 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 Bio-e 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.
 Kiernan MC, et al. Amyotrophic lateral sclerosis. The Lancet, 2011; 377(9769):942-955.
 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.
 Kiernan MC, et al. Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nat Rev Neurol 2021;17:104–118.
 Hulisz, Am J Manag Care. 2018;24:S320-S326,
 Vucic et al. Int J Mol Sci. 2019;20(11):2818;
 Morrison et al., Expert Rev Mol Med, 2006;8(11);
 Chio et al., Amyotroph Lateral SCler/. 2009; 10(5-6)