Treatments for Disease Modification in Development

Modification of genetic expression to enhance neuronal development and repair is a hot topic in the search for new therapies. Tofersen, an antisense oligonucleotide (ASO) that decreases SOD1 levels by inhibiting SOD1 mRNA translation, has shown some promise in clinical studies in people with ALS who have SOD1 mutations. In another form of hereditary motor neuron disease, spinal muscular atrophy (SMA), the use of ASOs has proved highly helpful. Reference: https://alstreatmentprotocol.blogspot.com/2021/10/treatments-for-disease-modification-in.html

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