MyFSHD

"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 side, we discuss a little more Avidity and Vita cell therapy.

"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.