MyFSHD copertina

MyFSHD

MyFSHD

Di: Peter L Jones PhD
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A proposito di questo titolo

 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 Economia Gestione e leadership Management
Episodi
  • All-in-one version of our evaluation of EPI-321 data and trial

    All-in-one version of our evaluation of EPI-321 data and trial
    Sep 14 2025

    "There's another old saying, Senator: Don't piss down my back and tell me it's raining." We know a lot of you don't have the time or interest in all the details so we packaged up the highlights of the gene therapy series, with some new information and evaluation, too, into one shorter (but not short enough, we know) episode.
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    1 ora e 36 min
  • Our evaluation and opinions of the EPI-321 clinical trial design and potential based on the pre-clinical data

    Our evaluation and opinions of the EPI-321 clinical trial design and potential based on the pre-clinical data
    Sep 6 2025

    "But did you bleed him? - Aesculapius says that bleeding is not a cure. It is only a way of proving that you are a physician at the expense of your patient." Finally, we discuss the EPI-321 epigenetic editing phase I/II clinical trial design and its potential for answering key questions necessary for going to phase III and for providing benefit to participants and patients.
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    46 min
  • Our scientific evaluation of and opinions on the EPI-321 publicly available preclinical data

    Our scientific evaluation of and opinions on the EPI-321 publicly available preclinical data
    Sep 6 2025

    "Good morning, and in case I don't see ya, good afternoon, good evening, and good night!" We take our time to evaluate publicly available preclinical data for EPI-321 used to support the Epicrispr Biotechnology first-in-human gene therapy clinical trial for FSHD. We discuss the data in the context of what is known from other neuromuscular disease gene therapy trials and integrate their own data in respect to what metrics are likely needed to be met for have any chance at providing benefit to patients.
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    2 ore e 4 min
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