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MyFSHD

MyFSHD

Von: Peter L Jones PhD
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 MyFSHD is about education and personal empowerment for the worldwide facioscapulohumeral muscular dystrophy (FSHD) community. Here we have discussions and commentary hosted by FSHD researcher Peter Jones, PhD, on many things of interest to the FSHD community. Learn about the science behind the different FSHD therapeutic approaches, FSHD pathology, family genetics and FSHD diagnostics. We will discuss upcoming clinical trials and what to look forward to. You will get to understand how you can be better prepared, become involved, and help contribute to defeating FSHD once and for all.Peter L Jones, PhD Management & Leadership Ökonomie
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  • Reddit Q&A (part 2). No "A", a little "I". Peter and Brad finish up the line of Reddit questions.

    Reddit Q&A (part 2). No "A", a little "I". Peter and Brad finish up the line of Reddit questions.
    May 9 2026

    "I'm not bad. I'm just drawn that way." Today we finish up some questions on clinical trials, therapeutics, what may be in the future, and other random FSHD topics. Good times :)
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    2 Std. und 4 Min.
  • Some clinical trial news of the day (March 27, 2026 - **updated March 29th**)

    Some clinical trial news of the day (March 27, 2026 - **updated March 29th**)
    Mar 29 2026

    "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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    1 Std. und 9 Min.
  • Reddit Q&A 3/17/2026 pt 1 (no song); Peter sits down with Brad our Angry Dad

    Reddit Q&A 3/17/2026 pt 1 (no song); Peter sits down with Brad our Angry Dad
    Mar 27 2026

    "I am the eyes and ears of this institution, my friends." You asked, we (try to) answer your questions. Part 1 of 2.

    There is no song in this version due to copyright issues causing it to be removed for some of our international listeners, otherwise, same as the original version.
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    1 Std. und 55 Min.
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