MyFSHD

Auteur(s): Peter L Jones PhD
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  • Résumé

  • 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
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Développement commercial et entrepreneuriat Gestion et leadership Économie
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Épisodes
  • For FSHD gene therapy you need to "Have-a-Little-Heart"

    For FSHD gene therapy you need to "Have-a-Little-Heart"
    Oct 11 2024

    "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.
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    32 min
  • Fulcrum follow-up with our Angry Dad

    Fulcrum follow-up with our Angry Dad
    Sep 28 2024

    "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.
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    1 h et 37 min
  • Our take on the REACH losmapimod clinical trial results

    Our take on the REACH losmapimod clinical trial results
    Sep 19 2024

    "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.
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    1 h et 58 min

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