Yi Dai

Stories

RNA activation shows first human proof-of-mechanism in Duchenne muscular dystrophy

Presented first-in-human data at WMS 2026

Three boys with Duchenne muscular dystrophy received monthly infusions of an experimental drug. Four months later, muscle biopsies showed 3.5- to 5.3-fold increases in utrophin, a protein that can stand in for the missing dystrophin.

Updated Oct 21

Notable Quotes

In the first three boys treated, we observed a complete and internally consistent chain of evidence: paired muscle biopsies showed utrophin robustly upregulated and correctly localized at the sarcolemma, muscle architecture improved with less fibrosis and fat infiltration, and motor and pulmonary measures held steady or improved over the same window.

from RNA activation shows first human proof-of-mechanism in Duchenne muscular dystrophy · Updated Oct 21