Épisodes

  • Pigs and p38
    Jun 1 2024

    "You're messing with the wrong guy!!!!" We expand a bit more on the utility of the FSHD-like pig model and then discuss the science behind and implications of p38 inhibition for FSHD.

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    1 h et 34 min
  • The three little (FSHD) piggies.
    Jan 28 2024

    "We don't get a lot of things to really care about." So, you think you are interested in science, eh? Well, let's see how the sausage is made. Today we share our experience generating the FSHD-like minipig models, which will be key tools for testing and advancing better FSHD therapeutics and developing methods for building back your muscles.

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    2 h et 11 min
  • CRISPR in the clinic
    Dec 24 2023

    "Come out to the coast, we'll get together, have a few laughs..." The holiday season we discuss the recent FDA approved CRISPR therapy for sickle cell disease and some dynamics of methylation.

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    59 min
  • Allow me to reintroduce Jaegerthekidd
    Nov 4 2023

    "As I leave my competition respirator style, climb the ladder to success escalator style." Today we have the Jaegercast, but first we continue our discussion about apabetalone, a new candidate drug for FSHD.

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    56 min
  • Assessing the case, so far, for apabetalone as a new drug candidate being investigated for FSHD.
    Nov 1 2023

    "I don't know how I'm going to live with myself if I don't stay true to what I believe." We evaluate the recent published work describing apabetalone, a small molecule drug from Resverlogix Corp that has been around for awhile and in clinic for other indications and is now being assessed more seriously as a potential therapeutic for FSHD. Overall, while lacking in some areas, this initial study is generally positive and supports that it is a new candidate worthy of further evaluation.

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    1 h et 24 min
  • Catching up on clinical trials
    Sep 24 2023

    "I am sick and tired of the entire western world knowing how my kidneys are functioning!" We discuss current and upcoming FSHD clinical trials and touch a little bit on funding.

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    1 h et 30 min
  • Live from Australia
    Sep 13 2023

    "Farmer Hoggett knew that little ideas that tickled, and nagged, and refused to go away should never be ignored, for in them lie the seeds of destiny." We are in Australia promoting FSHD awareness with Parliament and to gather government support for FSHD diagnostics and clinical trial infrastructure, as well as catching up with many of our Australian friends.

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    1 h et 14 min
  • A gene therapy approval for Duchenne muscular dystrophy and understanding Therapeutic Misconception.
    Jul 28 2023

    "Life has meaning only in the struggle. Triumph or defeat is in the hands of God. So let us celebrate the struggle!" With the FDA approval of the first gene therapy for DMD and clinical trials for FSHD in all stages of planning and performance, we take a moment to discuss the history of the DMD gene therapy path to approval as it relates to FSHD and address the important, yet oft ignored, concept of Therapeutic Misconception.

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    1 h et 57 min