Treatment of Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping
Not all patients with Duchenne muscular dystrophy share the same underlying mutation. In patients whose DMD mutation is amenable to exon 45 skipping, a genotype-directed treatment strategy applies — distinct from management in other DMD sub-populations.
Clinical Scenario
The patient carries a DMD mutation that is amenable to skipping exon 45. This specific genetic designation is the determining factor for eligibility for the targeted intervention described in this protocol.
Treatment Approach
Management in this sub-population involves a targeted antisense oligonucleotide exon-skipping therapy. The specific agent, complete dosing schedule, route, administration parameters, and monitoring guidance are detailed in the full structured protocol.
References
- Amondys 45 (casimersen) – An ASO indicated for treatment of patients with DMD amenable to skipping exon 45.
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