Treatment of Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping

Clinical Scenario

This protocol applies to patients diagnosed with Duchenne muscular dystrophy (DMD) whose underlying genetic mutation is amenable to exon 53 skipping — a specific subset of the DMD population identifiable through molecular genetic testing.

Treatment Approach

Management in this setting involves an antisense oligonucleotide (ASO) exon-skipping therapy specifically approved for the exon 53-amenable DMD population. The full treatment selection criteria, sequencing, and monitoring guidance are available in the complete protocol.

Full regimen details, including agent selection and clinical considerations, are in the structured protocol below.

Instant Access to Structured Evidence-Based Regimens

References

Viltepso (viltolarsen) — An ASO indicated for the treatment of patients with DMD amenable to skipping exon 53.
Vyondys 53 (golodirsen) — An ASO indicated for the treatment of patients with DMD amenable to skipping exon 53.

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