26 March 2026
We welcome the announcement from Sarepta Therapeutics on early clinical data from its siRNA program targeting FSHD.
These initial Phase 1/2 results demonstrate encouraging signs, including successful delivery of therapy to muscle tissue, early biomarker activity, and a favourable safety profile to date.
Importantly, this program is designed to directly target DUX4, the underlying driver of FSHD, representing a highly strategic approach to disease modification.
While these findings are early and further clinical validation is required, they contribute to growing momentum across the FSHD therapeutic landscape and reinforce the potential of RNA-based approaches.
For our community, this represents continued progress toward effective treatments and, ultimately, improved outcomes for people living with FSHD.
We look forward to following the advancement of this program and the broader pipeline with cautious optimism.