"Blue Horseshoe loves Anacott Steel." Today we cover recent clinical trial news in the FSHD space and delve a bit deeper into some topics from part 1 of our Reddit Q&A. **Updated version**The lowlight was Roche canceling their trial. The highlight was Sarepta's announcement of preliminary clinical data. Getting siRNAs, ASOs, and viruses into liver is easy because liver is filled with leaky capillaries, however, muscle is poorly vascularized and blood vessels have tight junctions so targeting cell receptors for enhanced transcytosis greatly enhances skeletal muscle delivery. Sarepta's targeting of the alpha v beta 6 integrin is a great advance in delivery of siRNA against DUX4 and, as predicted, shows the best DUX4 knockdown we have seen so far.
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Reddit Q&A 3/17/2026 pt 1 (no song); Peter sits down with Brad our Angry Dad
Our takes on recent (~Jan 2026) FSHD clinical trial updates
All-in-one version of our evaluation of EPI-321 data and trial
Our evaluation and opinions of the EPI-321 clinical trial design and potential based on the pre-clinical data
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