New hope for duchenne: targeted drug trial aims to slow muscle decline

NCT ID NCT07587242

First seen May 15, 2026 · Last updated May 15, 2026

Summary

This study tests an experimental drug called AOC 1044 (delpacibart zotadirsen) in 70 boys aged 7 to 16 with Duchenne muscular dystrophy who have a specific genetic change (exon 44 skipping). The drug is given through a vein and aims to improve muscle function, such as how fast they can stand up from a chair. Half the participants will receive the drug and half a placebo, and all will be followed for over a year to check safety and effectiveness.

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