『MyFSHD』のカバーアート

MyFSHD

MyFSHD

著者: Peter L Jones PhD
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今ならプレミアムプランが3カ月 月額99円

2026年5月12日まで。4か月目以降は月額1,500円で自動更新します。

概要

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 マネジメント マネジメント・リーダーシップ 経済学
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  • Some clinical trial news of the day (March 27, 2026 - **updated March 29th**)
    2026/03/29

    "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 時間 9 分
  • Reddit Q&A 3/17/2026 pt 1 (no song); Peter sits down with Brad our Angry Dad
    2026/03/27

    "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 時間 55 分
  • Our takes on recent (~Jan 2026) FSHD clinical trial updates
    2026/01/15

    "So, Mr Tipton, how could it take you five minutes to cook your grits when it takes the entire grit-eating world twenty minutes?" We discuss the initial Epicrispr Biotech press release pertaining to their EPI-321 phase 1/2 clinical trial and the big picture science behind the RESTEM phase 1/2 clinical trial in progress and the Scholar Rock upcoming phase 2 clinical trial.

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    46 分
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