MyFSHD

Follow MyFSHD
Share on
Copy link to clipboard

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


    • Apr 25, 2025 LATEST EPISODE
    • every other week NEW EPISODES
    • 1h 19m AVG DURATION
    • 96 EPISODES


    Search for episodes from MyFSHD with a specific topic:

    Latest episodes from MyFSHD

    Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 2)

    Play Episode Listen Later Apr 25, 2025 65:00


    "I gotta know what a 5 dollar shake tastes like." We continue our conversation with Tamara Gottlieb, who shares her years of experience helping to keep her family above the FSHD curve and improve quality of life. It's a lot of work, but it can help. Part 2 of 2. Tamara is one of the leaders (along with Emily Ca and Wayne Nesbit) of the "FSHD - supplements, nutrition, and peer support" Facebook gropou, so check it out for more information.

    Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 1)

    Play Episode Listen Later Apr 18, 2025 50:35


    "You are what you eat, and I'm freaking delicious!" Our guest today, Tamara Gottlieb, is the driving force for our First Family of FSHD Fitness, Nutrition, and Lifestyle, and she shares with us what she has learned over her years of experience helping keep her family above the FSHD curve and improve quality of life. It's a lot of work, but it can help. Part 1 of 2. Tamara (along with Emily Ca and Wayne Nesbit) is one of the leaders of the "FSHD - supplements, nutrition, and peer support" Facebook group, so check it out for more information.

    FaceToned® Exercises for the Face with Carme Farré

    Play Episode Listen Later Apr 12, 2025 66:28


    "Come on. No, no, don't do that. Don't give me the pouty batface." We have video today (!) for our special guest Carme Farré, who founded FaceToned®, and she shares her experiences as part of an FSHD family and how her techniques for facial fitness can help the FSHD community. If interested, you can arrange a free consultation below:

    Brad, our angry Dad with your questions (pt 2)

    Play Episode Listen Later Apr 3, 2025 64:33


    "Never go in against a Sicilian when death is on the line!" We have both survived so far and are back with part 2 of your Reddit questions on FSHD.

    Brad, our angry Dad is back with your questions (Pt 1)

    Play Episode Listen Later Mar 25, 2025 69:13


    "The battle of wits has begun. It ends when you decide and we both drink - and find out who is right, and who is dead." Today Brad, our angry dad, brings a list of your FSHD Reddit questions looking for answers.

    The science and the foundational funding leading to the current technologies in FSHD clinical trials.

    Play Episode Listen Later Mar 15, 2025 109:10


    "The most valuable commodity I know of is information." Today we discuss current clinical trials in FSHD and the foundational science behind the technology. While NIH has funded much of the foundational science that todays therapeutic approaches are built upon, it is the private individual and foundation support from those with vested interests in a rare disease that drive industry investment that gets the therapies over the line and into the clinic and ultimately to patients.

    Friends and funding.

    Play Episode Listen Later Feb 23, 2025 94:06


    "Good heavens, are you still trying to win? You've got an over developed sense of vengeance. It's going to get you into trouble someday." We remember Jenny Hasenjaeger and discuss research funding so that we don't let anyone else down.

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

    Play Episode Listen Later Oct 11, 2024 32:02


    "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.

    Fulcrum follow-up with our Angry Dad

    Play Episode Listen Later Sep 28, 2024 97:03


    "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.

    Our take on the REACH losmapimod clinical trial results

    Play Episode Listen Later Sep 19, 2024 118:20


    "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.

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

    Play Episode Listen Later Jun 22, 2024 75:45


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

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

    Play Episode Listen Later Jun 14, 2024 65:39


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Pigs and p38

    Play Episode Listen Later Jun 1, 2024 93:51


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    The three little (FSHD) piggies.

    Play Episode Listen Later Jan 28, 2024 130:57


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    CRISPR in the clinic

    Play Episode Listen Later Dec 24, 2023 58:41


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Allow me to reintroduce Jaegerthekidd

    Play Episode Listen Later Nov 4, 2023 55:39


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Assessing the case, so far, for apabetalone as a new drug candidate being investigated for FSHD.

    Play Episode Listen Later Nov 1, 2023 84:03


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Catching up on clinical trials

    Play Episode Listen Later Sep 24, 2023 89:41


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Live from Australia

    Play Episode Listen Later Sep 13, 2023 74:10


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    A gene therapy approval for Duchenne muscular dystrophy and understanding Therapeutic Misconception.

    Play Episode Listen Later Jul 28, 2023 117:02


    "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. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    More questions, more answers, and some explaining to do.

    Play Episode Listen Later Jun 20, 2023 97:19


    "It's human nature to lie. Most of the time we can't even be honest with ourselves." At MyFSHD is is always FSHD Day. We continue the conversation around therapeutic modalities and clinical trials, hopefully providing additional context for clarification, or just digging a deeper hole. You tell us. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Your questions, our answers.

    Play Episode Listen Later Jun 10, 2023 87:04


    "Apes don't read philosophy." "Yes they do, Otto, they just don't understand it!" We're here to answer your questions and help you understand all things (FSHD) on your mind. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Live from the Biologic Scaffolds for Regenerative Medicine Symposium in Napa California

    Play Episode Listen Later May 27, 2023 101:56


    "We can rebuild him. We have the technology. We can make him better than he was. Better, stronger, faster." Today we are at the Biologic Scaffolds for Regenerative Medicine Symposium to discuss novel ways to potentially help FSHDers maintain strength and slow down pathology. Additional technology being presented by be applicable to help regain muscle mass after therapy. Joining us is one of the best FSHD advocates around, Emma Weatherley from FSHD Global Research Foundation in Australia. And we drop the newest track from Jaeger the Kid! --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    We are back with updates on our worldwide efforts for FSHD diagnostics.

    Play Episode Listen Later May 17, 2023 65:13


    "We Are Unique, Gentlemen, In That We Create Ourselves Through Long Years Of Rigorous Training, Sacrifice, Denial, Pain." Today we focus on our worldwide efforts to help FSHDers everywhere. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Roundup of the FSHD news of the day with our CRISPR Goddess.

    Play Episode Listen Later Apr 12, 2023 92:28


    Roy: "This is everything, ain't it? This is the choice it comes down to - this is our immortality." Romeo: "You don't need to be thinking immortality - you need to be thinking hit the 7 iron!" Dr Charis Himeda joins us to discuss recent news on stem cells, losmapimod, gene therapy, and antisense for FSHD. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    The return of Brad (the angry dad).

    Play Episode Listen Later Mar 26, 2023 111:18


    "You just put your pickle on everybody's plate, college boy, and leave the hard stuff to me." We talk a little more about funding, hopefully providing some clarity, then bring on our Angry Dad for some questions, and then bring it home with another new track from Jaeger. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Rare Disease Day and Accountability

    Play Episode Listen Later Mar 10, 2023 100:40


    "Sorry, Vern. I guess a more experienced shopper could have gotten more for your seven cents." Rare disease day is the last day of February, which got us thinking about what really needs to be done to get over the line. We also answer listener questions about animal models and the Avidity, myostatin inhibition, and cell therapy trials. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Reintroducing the MyFSHD podcast

    Play Episode Listen Later Feb 25, 2023 89:50


    "Come on in, grab a beer. Don't cost nothing." We have been podcasting about all things FSHD and realize that we have a lot of new listeners over the past year so we want to take this opportunity to catch everyone up on what the MyFSHD podcast is all about. You may have noticed that we are a bit different. We are not asking for money and we are not selling you anything, just real talk from experts in all things FSHD to help you understand and navigate the space with knowledge. On the science side, we discuss a little more Avidity and Vita cell therapy. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    "A" is for Avidity

    Play Episode Listen Later Feb 13, 2023 73:58


    "Why, sometimes I've believed as many as six impossible things before breakfast." Experimental therapies designed specifically for FSHD are finally arriving for trial in the clinic (i.e., in people). Today we discuss the upcoming Phase 1/2 clinical trial from Avidity using their antibody oligo conjugated siRNA designed specifically to knockdown the DUX4 mRNA in FSHD. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    We have a lot in common with pigs. "Don't flatter yourselves", reply the pigs :).

    Play Episode Listen Later Feb 6, 2023 87:34


    "Do you ever have déjà vu, Mrs. Lancaster?" "I don't think so, but I could check with the kitchen" We will discuss some cool new pig data and how we are going about making sure our FSHD-like minipig models will be made right and properly characterized to be useful for testing FSHD therapeutics and muscle building strategies. As you know, the devil is always in the details and so far pigs are looking pretty darn good! --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Our "State of the Field" address.

    Play Episode Listen Later Jan 27, 2023 107:15


    "I've been going to this high school for 7-1/2 years. I'm no dummy." As 2023 gets underway, let's check in and see where the field stands - in our opinion, of course. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Happy 2023 from MyFSHD!

    Play Episode Listen Later Jan 15, 2023 92:04


    "Life isn't like in the movies. Life..... is much harder." Whatever you end up doing, love it. And we love working every day on the problem of FSHD, educating others about the science, helping people learn about themselves, and informing the community of advancements. We're back and going strong, starting Season 2 with a discussion on the future of clinical trials and therapies, as requested by you. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message

    Merry Christmas, Happy Hanukkah, and Happy Holidays from MyFSHD

    Play Episode Listen Later Dec 25, 2022 105:44


    "Remember no man is a failure who has friends." We catch up with a few friends in this holiday season as FSHD research plows ahead. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Antisense, gene therapy, and stem cell news, and the Blues Brothers

    Play Episode Listen Later Dec 15, 2022 86:04


    "I want four fried chickens and a Coke". Dr. Charis Himeda joins us as we discuss some encouraging recent data from Sarepta for one of their DMD gene therapy trials and from Avidity for their myotonic dystrophy phase I/II trial and how it all potentially impacts FSHD. In addition, we revisit the potential (or lack there of) for placental or umbilical cord derived stem cell therapy for FSHD. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    The best humanized FSHD muscle xenograft mouse model with Dr. Bob Bloch.

    Play Episode Listen Later Dec 2, 2022 114:48


    "Never mind manoeuvres, always go at them". Dr. Bob Bloch from the University of Maryland School of Medicine joins us to discuss his development of the human FSHD muscle xenograft mouse model that is a key tool in the pre-clinical testing pipeline for FSHD therapeutics and biomarker discovery. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    We have a lot to be thankful for!

    Play Episode Listen Later Nov 24, 2022 116:55


    "You're messing with the wrong guy!" Happy Thanksgiving to our friends in the US, and for everyone else, it is always a good time to remember and give thanks for those you care about and who care about you. For us, that is the worldwide FSHD community. Today we have Brad, our angry dad, and discuss accessibility issues for safety and dealing with roadblocks in research. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    More on FSHD research testing, CRISPR, and minipigs

    Play Episode Listen Later Nov 20, 2022 97:56


    “The most terrifying day of your life is the day the first one is born. Your life as you know it is gone … But they learn how to walk, and they learn how to talk, and you want to be with them. And they turn out to be the most delightful people you will ever meet in your life.” We understand many of you participate in our FSHD research testing to learn more about yourselves and especially at risk family members. Here we discuss more about the procedure and results and interpretations. In addition, some more on CRISPR approaches to FSHD and a short FSHD minipig update. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Catch up on some news of the week then sit down with FSHD researcher Maryam Farooqi

    Play Episode Listen Later Nov 10, 2022 97:34


    "Seek not to know the answers, but to understand the questions." Get to know Maryam Farooqi of the Jones Lab for FSHD. She is the first half of the team that performs all the FSHD research testing and is key for a number of other FSHD projects (i.e. FSHD-like mice and minipig projects). --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    News of the week on Fulcrum and CRISPR gene therapy.

    Play Episode Listen Later Nov 6, 2022 73:36


    "Advertising has us chasing cars and clothes, working jobs we hate so we can buy s--- we don't need." We discuss the publicly available data for the Fulcrum Open Label Extension of the ReDUX4 trial and the recent CRISPR-activation "N-of-one" trial in DMD and what that means for FSHD gene therapy. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Happy Nevada Day! Today we talk about early onset FSHD and getting adolescents into clinical trials.

    Play Episode Listen Later Oct 31, 2022 105:40


    "I would like, if I may, to take you on a strange journey." Ally Roets and Kristin Zwickau from the Early Onset FSHD Parent Group join us today to discuss what we need to do to get trial ready for those under 18 years old. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Monopoly money in science and to test, or not to test, (your kids) for FSHD

    Play Episode Listen Later Oct 21, 2022 95:43


    "Roads? Where we're going, we don't need roads." We address listener questions on the cost of research and bringing new therapeutics to market and the debate over testing kids for FSHD when they are in an FSHD family but don't show any overt symptoms. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Keeping up with even more investment and more technology coming into the FSHD space.

    Play Episode Listen Later Oct 13, 2022 74:10


    "Was it over when the Germans bombed Pearl Harbor? Hell no!" We're just getting started at tearing this disease down, with more money being invested and more companies getting into the space seemingly every week. Jaeger drops another new track as we're trying to keep you up to speed with developments with Fulcrum, Solve FSHD, Vita Therapeutics, and more. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    More clinical trial discussion with our FSHD Mom and Dad.

    Play Episode Listen Later Oct 5, 2022 109:39


    "Just how bad is it?" "It's a fire. All fires are bad." We sit down with our FSHD Mom and fire inspector Dad and talk FSHD clinical trials. Also, it's fire prevention month, check your smoke and carbon monoxide detectors, make a plan in case of fire, know your two exits, have a place to meet, and make sure to discuss your plan with your family and/or roommates. Let's make sure we all make it to see this thing through to a cure. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Update on clinical trials for FSHD

    Play Episode Listen Later Oct 1, 2022 73:48


    "Oh, you should never, never, doubt what nobody is sure about." Fulcrum has the losmapimod phase III trial going and now both Roche and Avidity just announced their upcoming clinical trials for FSHD. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Catching up on FSHD, Saturday Sept 24th, 2022

    Play Episode Listen Later Sep 24, 2022 85:26


    “Well, I guess if a person never quit when the going got tough, they wouldn't have anything to regret for the rest of their life." No regrets here, no matter how tough, we'll never quit till the job is done. Today we finish up on the Chocolate Ball, talk more on Dyne's announcement, and go back over DNA methylation as a diagnostic and prognostic for FSHD. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Live from Sydney, Australia, it's MyFSHD!

    Play Episode Listen Later Sep 17, 2022 83:54


    "That's not a knife...... that's a knife!" Drs. Peter and Takako Jones attend the 11th Annual Sydney Chocolate Ball to raise funds for FSHD Global Research Foundation, but we still talk about FSHD news of the day, which is DNA methylation (no surprise) and a big disappointment from Dyne Therapeutics. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Brunch with Maryam (and Takako and Peter). Our 50th episode!

    Play Episode Listen Later Sep 11, 2022 89:29


    "Faced with overwhelming odds I'm going to have to science the s*** out of this." We celebrate our 50th podcast in style with Maryam Farooqi taking over the hosting duties (and the kitchen) as we discuss all things FSHD. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    FSHD catch-up, September 7, 2022

    Play Episode Listen Later Sep 8, 2022 63:44


    "The secret's in the sauce." We discuss some recent news in the neuromuscular disease space and FSHD publications on cell therapy and biomarkers. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    More CRISPR questions from the audience.

    Play Episode Listen Later Sep 1, 2022 79:58


    "Your scientists were so preoccupied with whether they could, they didn't stop to think if they should." Our CRISPR Goddess Charis talks with host Peter and answers your questions on CRISPR, eventually. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Kari Cilliers, a medical student from South Africa found her way to Nevada to learn about FSHD

    Play Episode Listen Later Aug 27, 2022 69:58


    "She made me realize just how precious wild places are." Our visiting medical student from South Africa, and fellow FSHDer, Kari Cilliers, joins Dr. Jones to discuss her time in the US working on FSHD. --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Our FSHD Dad is back with more questions, comments and concerns on biomarkers, clinical trials, MRI, and supplements

    Play Episode Listen Later Aug 19, 2022 109:51


    "If you have one bucket that contains 7 gallons and one bucket that contains 2 gallons, how many buckets do you have?" Our FSHD dad asks questions on research and funding priorities, biomarkers, MRI, and what we are doing as a field to make sure we know if a drug works or not in trial. Plus, the world premiere of a new track from Jaeger! --- Send in a voice message: https://anchor.fm/peter-l-jones/message

    Claim MyFSHD

    In order to claim this podcast we'll send an email to with a verification link. Simply click the link and you will be able to edit tags, request a refresh, and other features to take control of your podcast page!

    Claim Cancel