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  • Our scientific evaluation of and opinions on the EPI-321 publicly available pre-clinical data
    2025/09/06

    "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 時間 4 分
  • Prequel for the Epicrispr Bio data and clinical trial discussion
    2025/09/06

    "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 時間 23 分
  • The nuts and bolts of gene therapy for FSHD (Part 2 of 2)
    2025/08/17

    "...and by the time they figure out what went wrong, we'll be sitting on a beach, earning twenty percent." This is part 2 of our podcast series covering the important factors in successful gene therapy and introduces the knowledge gained from other neuromuscular diseases and how it can be applied to positively impact gene therapy in FSHD. In part 2, we focus on viral dosing and how it affects therapeutic biodistribution and expression. In addition, we reveal what is known about variable durability of transgene expression and what to look for to predict a durable, or non-durable, therapeutic. Check out the MyFSHD Youtube for the music, as well.

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    1 時間 34 分
  • The nuts and bolts of gene therapy for FSHD (Part 1 of 2)
    2025/08/15

    "My friends are toys. I make them. It's a hobby. I'm a genetic designer." This is a two part podcast covering the important factors is successful gene therapy and introduces the knowledge gained from other neuromuscular diseases and how it can be used to positively impact gene therapy in FSHD. In part 1, we focus on AAV serotypes, transgene constructs, immune responses, and delivery. We look into DMD gene therapy and how that can impact the current FSHD gene therapy programs. Check us out on the MyFSHD Youtube channel as well.

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    1 時間 20 分
  • Part 2 of our discussion with our teen FSHD advocates
    2025/07/30

    "You were seeking some information of some kind?" Continuing our discussion with our young FSHD advocates, asking and answering some questions.

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    42 分
  • Teens promoting their "Cure FSHD for All" initiative, podcast part 1
    2025/07/28

    "And that would tell you what is obvious right now? That avoiding apple skins and pizza has no effect on this brutal disease?" Today we discuss the Cure FSHD for All (go to: www.curefshdforall.com) initiative for inclusion of FSHDers under 18 and those with limited mobility into clinical trials and for access to developing therapies.

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    1 時間 19 分
  • MyFSHD is worldwide on FSHD Day
    2025/06/24

    "You're gonna need a bigger boat." MyFSHD is our summer blockbuster passion project. We discuss how to get it done, whatever your "it" is, in the FSHD space, as well as our trip to Shanghai and Hanoi to continue our efforts to help FSHDers around the world.

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    1 時間 19 分
  • Our take on Avidity's FORTITUDE phase 1/2 trial data
    2025/06/11

    "I'll have what she's having." We'll take you through and discuss the recent release of Avidity Biosciences topline results from the phase 1/2 FORTITUDE trial and the preliminary data from the biomarker cohort extension.

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    55 分