MyFSHD Podcast Por Peter L Jones PhD arte de portada

MyFSHD

MyFSHD

De: 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 Economía Gestión Gestión y Liderazgo
Episodios
  • 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 m
  • Our scientific evaluation of and opinions on the EPI-321 publicly available pre-clinical data

    Our scientific evaluation of and opinions on the EPI-321 publicly available pre-clinical 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 h y 4 m
  • Prequel for the Epicrispr Bio data and clinical trial discussion

    Prequel for the Epicrispr Bio data and clinical trial discussion
    Sep 6 2025

    "In the history of science, innovative concepts occasionally arise from sudden left-field inspiration." Here we make sure everyone is up to speed on the types of CRISPR inhibition for FSHD and FSHD epigenetics s that we are all ready to evaluate the preclinical data supporting the Epicrispr Biotechnology EPI321 clinical trial.
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    1 h y 23 m
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