"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.
"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.
"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 sid...
"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.
"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!
"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.
"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.
"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.
"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.
"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.
"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.
“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...
"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).
"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.
"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.
"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.
"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.
"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.
"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.
“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.
"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.
"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.
"The secret's in the sauce." We discuss some recent news in the neuromuscular disease space and FSHD publications on cell therapy and biomarkers.
"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.
"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.
"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!
“Invention, my dear friends, is 93% perspiration 6% electricity 4% evaporation and 2% butter scotch ripple.” Natalie Cooney and Emma Weatherley sit down with MyFSHD to talk about how initiatives from the FSHD Global Research Foundation are helping the FSHD community in Australia and around the world, and also the upcoming Sydney Chocolate Ball.
"Who's gonna turn down a Junior Mint? It's chocolate, it's peppermint..... it's delicious!" Tamara and Michael Gottlieb join us to discuss how nutrition, vitamins, dietary supplements, and lifestyle adjustments have greatly improved their muscle health in the FSHD and non-FSHD members of their family. Tamara is one of the founders of the "FSHD - supplements, nutrition, and peer support" Facebook group.
"The creatures outside looked from pig to man, and from man to pig, and from pig to man again; but it already was impossible to say which was which." Peter talks a bit more about the FSHD-like minipigs being developed for therapeutics and muscle regeneration.
"Go the distance." Jenny Hasenjaeger of MyFSHD sits down with Ben Brittain to discuss his FSHD and what he intends to do about it.
