Tofersen - A Ray of Hope for ALS Patients : A Mini Review

Abstract

Despite thorough investigation, amyotrophic lateral sclerosis (ALS) continues to be adegenerative neurological condition that is inevitably fatal and progresses over time. The limitedunderstanding of the fundamental causes of ALS has posed challenges in addressing the initialbiological processes of the disease, resulting in therapeutic interventions typically being appliedat later stages of the illness.The existence of genetic variants in ALS presents a distinctive chance for advancing therapeuticapproaches, as studying genetic associations could unveil valuable information about the causesof the disease. In the case of genetic ALS, there is a possibility to explore early intervention byidentifying individuals who are at risk and have not yet shown symptoms but possess specificgenetic variations that contribute to the condition. Tofersen, an oligonucleotide, has beenspecifically created to diminish the production of superoxide dismutase 1 (SOD1) protein bybreaking down SOD1 mRNA.