Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis.

ABSTRACT: Aim: Phosphorodiamidate morpholino oligomers (PMOs) are exon-skipping therapies administered through once-weekly intravenous infusions used to treat Duchenne muscular dystrophy (DMD). This study assessed treatment patterns among patients with DMD receiving PMOs using administrative claims data while accounting for limitations in claims data for these therapies. Materials & methods: This study used Inovalon^® public and private closed claims data (1 June 2016-31 March 2024). Male patients with ≥1 claim for a PMO approved for DMD in the US (eteplirsen, casimersen, golodirsen and viltolarsen) were included. Index date was the first PMO claim. All available follow-up data were used to assess continuous PMO claims coverage, ≥60-day and ≥30-day gaps in PMO claims and PMO

SUBMITTER: Grabich S 

PROVIDER: S-EPMC12308544 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature