“Tofersen - A Ray of Hope for ALS Patients” : A Mini Review
Abstract
Despite thorough investigation, amyotrophic lateral sclerosis (ALS) continues to be a
degenerative neurological condition that is inevitably fatal and progresses over time. The limited
understanding of the fundamental causes of ALS has posed challenges in addressing the initial
biological processes of the disease, resulting in therapeutic interventions typically being applied
at later stages of the illness.
The existence of genetic variants in ALS presents a distinctive chance for advancing therapeutic
approaches, as studying genetic associations could unveil valuable information about the causes
of the disease. In the case of genetic ALS, there is a possibility to explore early intervention by
identifying individuals who are at risk and have not yet shown symptoms but possess specific
genetic variations that contribute to the condition. Tofersen, an oligonucleotide, has been
specifically created to diminish the production of superoxide dismutase 1 (SOD1) protein by
breaking down SOD1 mRNA.
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References
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