Summarized by Masters of Longevity from PR Newswire.
Clinical data show a systemically delivered small activating RNA increased a therapeutic muscle protein and improved tissue features in Duchenne muscular dystrophy patients.
Key Takeaways
- Paired muscle biopsies from three treated boys showed robust utrophin upregulation and correct sarcolemma localization.
- Morphometry at Day 113 reported mean myofiber cross-sectional area increases of 5% to 15% and diameter gains of 5.34 μm to 27.50 μm.
- Muscle fat fraction fell by 3% to 10% and motor and pulmonary measures were stable or improved through Day 169.
