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Brand: Amondys 45
Casimersen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. It belongs to a class of drugs known as morpholinos, which modify gene expression by binding to specific RNA sequences. Casimersen specifically targets exon 45 of the DMD gene pre-mRNA, leading to its exclusion during splicing, resulting in a partially functional dystrophin protein.
Casimersen is used to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 45 skipping.
Outcome:
Mechanism:
Most likely new formulation: Improved delivery methods or combination therapies (Year: 2026, 70% confidence)
Based on current usage trends and clinical trial data, there is a high likelihood of continued approval for Casimersen in the DMD patient population amenable to exon 45 skipping.
Antisense Oligonucleotide
Morpholino Oligomer