MyFSHD

Peter L Jones, PhD
  • 5,0 (4)
  • PHI LỢI NHUẬN
  • HẰNG NGÀY
MyFSHD

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. 11 THG 10

    For FSHD gene therapy you need to "Have-a-Little-Heart"

    "You know the Greeks didn't write obituaries. They only asked one question after a man died: 'Did he have passion?'. " Today we are joined by our CRISPR Goddess to discuss her new FSHD optimized gene therapy cassette that is stronger, safer, more compact and all around better than anything out there for use in FSHD and neuromuscular gene therapy approaches.

    32 phút

  2. 28 THG 9

    Fulcrum follow-up with our Angry Dad

    "Show me the money!" Brad our Angry Dad sits down with us and asks the questions on his mind about the Fulcrum results and others going forward.

    1 giờ 37 phút

  3. 19 THG 9

    Our take on the REACH losmapimod clinical trial results

    "I always tell the truth, even when I lie." Today we are joined by our CRISPR Goddess and cover the Fulcrum Therapeutics REACH phase 3 clinical trial data release and discuss our journey with Fulcrum from the beginning. While very disappointing from several aspects, in the end a lot of benefit truly has been gained for the FSHD field and we are grateful for Fulcrum's contributions to help others going forward. And there are many others coming along, this is the beginning and not the end for FSHD therapeutics.

    1 giờ 58 phút

  4. 22 THG 6

    A FORTITIDE follow-up with our (not so) Angry Dad.

    "There's a time for daring and there's a time for caution, and a wise man understands which is called for." Brad our Angry Dad is back with some questions for us on the Avidity FORTITUDE AOC 1020 phase 1/2 trial interim data report and some muscle building.

    1 giờ 16 phút

  5. 14 THG 6

    A(+) is still for Avidity: give me the good stuff!

    "Whatchyou talkin' 'bout Willis?" In a field where every press release is met with unsubstantiated hoopla and proclamations of BREAKTHROUGH! to fire you up to shake you down, we finally have some real news. We provide our evaluation of Avidity's public interim report on their FORTITUDE AOC-1020 phase 1/2 trial.

    1 giờ 6 phút

  6. 1 THG 6

    Pigs and p38

    "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.

    1 giờ 34 phút
  7. The three little (FSHD) piggies.

    28 THG 1

    The three little (FSHD) piggies.

    "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.

    2 giờ 11 phút

  8. 24/12/2023

    CRISPR in the clinic

    "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.

    59 phút
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Xếp Hạng & Nhận Xét

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/5
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Giới Thiệu

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.

Thông Tin

  • Nhà sáng tạo
    Peter L Jones, PhD
  • Năm hoạt động
    2022 - 2024
  • Tập
    89
  • Xếp hạng
    Sạch
  • Bản quyền
    © Peter L Jones, PhD
  • Trang web chương trình
    MyFSHD

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