MyFSHD

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.

  1. 7月4日

    Cleaning the lipstick off a pig

    "What is magic? Magic is deception, but deception designed to delight, to entertain, to inspire. It is about belief. Faith." Today we discuss the recent exciting data presented by Epicrispr and their EPI-321 gene therapy at the IRC , and more importantly, we discuss what they (shockingly) did not say, present, or disclose. Hmmmm..... While it is inconceivable (to us) that a company would not test if their specific treatment was actually working, it is equally unconscionable that a company (with the help of their foundation partners) would knowingly mislead the community, so we know that is not happening, but we do have some questions and look forward to clarification and real validation in the future.

  2. 6月21日

    World FSHD Day 2026

    "It's not who I am underneath, but what I do that defines me." We celebrate World FSHD Day with those that are making a difference in the world (and our fathers -- Happy Father's Day!) and then discuss what we would like to hear from the FSHD clinical trial companies at the upcoming FSHD International Research Congress.

  3. 5月17日

    Evaluating gene expression data in FSHD and presenting at ASGCT

    "I think the question we should be asking is, do you believe in this thing or not?" We discuss the value of MRI-informed muscle biopsies compared with uninformed biopsies, how we go about interpreting FSHD clinical trial gene expression data, and after the song, we show the Jones Lab CRISPRi presentation from the ASGCT annual meeting in Boston.

  4. 5月9日

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

    "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 :)

  5. 3月29日

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

    "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.

  6. 3月27日

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

    "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.

  7. 1月15日

    Our takes on recent (~Jan 2026) FSHD clinical trial updates

    "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.

  8. 2025/10/25

    More questions and comments with Brad, our Angry Dad

    "That's super frustrating. It just makes me wanna pick up whoever is standing closest to me and throw them through this window and out into the infinite abyss of nothingness!" Brad, our angry Dad, is back with some questions about clinical trials and muscle building.

すべて見る（114）

番組について

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.

情報

  • クリエイター
    Peter L Jones, PhD
  • 配信期間
    22年 - 26年
  • エピソード
    114
  • 制限指定
    不適切な内容を含まない
  • Copyright
    © Peter L Jones, PhD
  • 番組のWebサイト
    MyFSHD