Casimersen (AMONDYS 45™) is a therapeutic antisense oligonucleotide designed for patients with Duchenne Muscular Dystrophy (DMD) whose gene mutation is amenable to exon 45 skipping. DMD is a genetic disorder characterized by the absence of dystrophin, a crucial protein for muscle function. This results in progressive muscle degeneration and loss of mobility, eventually affecting the heart and lungs. Casimersen works by binding to the mutated pre-mRNA, skipping exon 45 to …

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Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disorder characterized by motor neuron degeneration, often leading to death within a few years. While there are limited treatments, genetic mutations in superoxide dismutase 1 (SOD1) have been identified in 2% of ALS cases. Tofersen, an antisense oligonucleotide (ASO), specifically targets SOD1 mRNA, reducing the production of toxic SOD1 protein and has shown promise in clinical trials, resulting in FDA approval in …

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