PODCAST · business
MyFSHD
by Peter L Jones, PhD
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.
Episodes
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FSHD Biopharma --Please include us in your clinical trials (or at least allow expanded access!)
"Houston, we have a problem". While it is exciting for the FSHD community that clinical trials are here and more are coming soon, the system is not working for everyone. Our young and non-ambulatory FSHDers of all ages are currently being left behind. Today, Maggie and Kate from the Cure FSHD for...
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Understanding the Satellos drug going to clinic for FSHD
"You're gonna need a bigger boat!" Today we discuss the third compound going to clinic emerging from Neil Camarta's "Shark Tank" meetings. This one from Satellos was designed to address the regeneration deficit in DMD caused by reduced asymmetric muscle stem cell division, and is now being...
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FSHD Phase I/II clinical trial previews for Scholar Rock, Soufflé and Dyne.
"If you want something bad, you have to fight for it. Step up you game, Scott. Break out the L-word". Today we discuss a little more on the Epicrispr clinical trial and preview the upcoming Scholar Rock, Soufflé and Dyne phase I/II clinical trials. As you know, the Devil is in the details. We are...
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Cleaning the lipstick off a pig
"What is magic? Magic is deception, but deception designed to delight, to entertain, to inspire. It is about belief. Faith." Today we discuss the recent exciting data presented by Epicrispr and their EPI-321 gene therapy at the IRC , and more importantly, we discuss what they (shockingly) did not...
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World FSHD Day 2026
"It's not who I am underneath, but what I do that defines me." We celebrate World FSHD Day with those that are making a difference in the world (and our fathers -- Happy Father's Day!) and then discuss what we would like to hear from the FSHD clinical trial companies at the upcoming FSHD...
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Evaluating gene expression data in FSHD and presenting at ASGCT
"I think the question we should be asking is, do you believe in this thing or not?" We discuss the value of MRI-informed muscle biopsies compared with uninformed biopsies, how we go about interpreting FSHD clinical trial gene expression data, and after the song, we show the Jones Lab CRISPRi...
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Reddit Q&A (part 2). No "A", a little "I". Peter and Brad finish up the line of Reddit questions.
"I'm not bad. I'm just drawn that way." Today we finish up some questions on clinical trials, therapeutics, what may be in the future, and other random FSHD topics. Good times :)
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Some clinical trial news of the day (March 27, 2026 - **updated March 29th**)
"Blue Horseshoe loves Anacott Steel." Today we cover recent clinical trial news in the FSHD space and delve a bit deeper into some topics from part 1 of our Reddit Q&A. **Updated version** The lowlight was Roche canceling their trial. The highlight was Sarepta's announcement of preliminary…
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Reddit Q&A 3/17/2026 pt 1 (no song); Peter sits down with Brad our Angry Dad
"I am the eyes and ears of this institution, my friends." You asked, we (try to) answer your questions. Part 1 of 2. There is no song in this version due to copyright issues causing it to be removed for some of our international listeners, otherwise, same as the original version.
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Our takes on recent (~Jan 2026) FSHD clinical trial updates
"So, Mr Tipton, how could it take you five minutes to cook your grits when it takes the entire grit-eating world twenty minutes?" We discuss the initial Epicrispr Biotech press release pertaining to their EPI-321 phase 1/2 clinical trial and the big picture science behind the RESTEM phase 1/2...
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More questions and comments with Brad, our Angry Dad
"That's super frustrating. It just makes me wanna pick up whoever is standing closest to me and throw them through this window and out into the infinite abyss of nothingness!" Brad, our angry Dad, is back with some questions about clinical trials and muscle building.
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All-in-one version of our evaluation of EPI-321 data and trial
"There's another old saying, Senator: Don't piss down my back and tell me it's raining." We know a lot of you don't have the time or interest in all the details so we packaged up the highlights of the gene therapy series, with some new information and evaluation, too, into one shorter (but not...
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Our evaluation and opinions of the EPI-321 clinical trial design and potential based on the pre-clinical data
"But did you bleed him? - Aesculapius says that bleeding is not a cure. It is only a way of proving that you are a physician at the expense of your patient." Finally, we discuss the EPI-321 epigenetic editing phase I/II clinical trial design and its potential for answering key questions necessary...
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Our scientific evaluation of and opinions on the EPI-321 publicly available preclinical data
"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…
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Prequel for the Epicrispr Bio data and clinical trial discussion
"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...
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The nuts and bolts of gene therapy for FSHD (Part 2 of 2)
"...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...
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The nuts and bolts of gene therapy for FSHD (Part 1 of 2)
"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...
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Part 2 of our discussion with our teen FSHD advocates
"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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Teens promoting their "Cure FSHD for All" initiative, podcast part 1
"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…
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MyFSHD is worldwide on FSHD Day
"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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Our take on Avidity's FORTITUDE phase 1/2 trial data
"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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Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 2)
"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…
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Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 1)
"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...
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FaceToned® Exercises for the Face with Carme Farré
"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...
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Brad, our angry Dad with your questions (pt 2)
"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.
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Brad, our angry Dad is back with your questions (Pt 1)
"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.
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The science and the foundational funding leading to the current technologies in FSHD clinical trials.
"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...
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Friends and funding.
"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.
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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...
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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.
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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...
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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.
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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...
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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.
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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...
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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.
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Allow me to reintroduce Jaegerthekidd
"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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Assessing the case, so far, for apabetalone as a new drug candidate being investigated for FSHD.
"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...
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Catching up on clinical trials
"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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Live from Australia
"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...
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A gene therapy approval for Duchenne muscular dystrophy and understanding Therapeutic Misconception.
"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...
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More questions, more answers, and some explaining to do.
"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.
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Your questions, our answers.
"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.
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Live from the Biologic Scaffolds for Regenerative Medicine Symposium in Napa California
"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...
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We are back with updates on our worldwide efforts for FSHD diagnostics.
"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.
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Roundup of the FSHD news of the day with our CRISPR Goddess.
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...
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The return of Brad (the angry dad).
"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.
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Rare Disease Day and Accountability
"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,...
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Reintroducing the MyFSHD podcast
"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...
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"A" is for Avidity
"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...
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ABOUT THIS SHOW
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.
HOSTED BY
Peter L Jones, PhD
CATEGORIES
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Frequently Asked Questions
How many episodes does MyFSHD have?
MyFSHD currently has 116 episodes available on PodParley. New episodes are automatically indexed when they're published to the podcast feed.
What is MyFSHD about?
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...
How often does MyFSHD release new episodes?
MyFSHD publishes regularly with 116 episodes available. Check the episode list for the typical release schedule.
Where can I listen to MyFSHD?
You can listen to MyFSHD on PodParley by clicking any episode. We provide an embedded audio player for direct listening, and you can also subscribe via your preferred podcast app using the RSS feed.
Who hosts MyFSHD?
MyFSHD is created and hosted by Peter L Jones, PhD.
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