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MyFSHD

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.
Currently playing episode

The three little (FSHD) piggies.

MyFSHDJan 28, 2024

00:00
02:10:57
The three little (FSHD) piggies.

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 developing methods for building back your muscles.

Jan 28, 202402:10:57
CRISPR in the clinic

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.

Dec 24, 202358:42
Allow me to reintroduce Jaegerthekidd

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.

Nov 04, 202355:40
Assessing the case, so far, for apabetalone as a new drug candidate being investigated for FSHD.

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 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.

Nov 01, 202301:24:03
Catching up on clinical trials

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.

Sep 24, 202301:29:41
Live from Australia

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 infrastructure, as well as catching up with many of our Australian friends.

Sep 13, 202301:14:09
A gene therapy approval for Duchenne muscular dystrophy and understanding Therapeutic Misconception.

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 gene therapy path to approval as it relates to FSHD and address the important, yet oft ignored, concept of Therapeutic Misconception.

Jul 28, 202301:57:01
More questions, more answers, and some explaining to do.

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.

Jun 20, 202301:37:19
Your questions, our answers.

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.

Jun 10, 202301:27:04
Live from the Biologic Scaffolds for Regenerative Medicine Symposium in Napa California

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 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!

May 27, 202301:41:56
We are back with updates on our worldwide efforts for FSHD diagnostics.

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.

May 17, 202301:05:13
Roundup of the FSHD news of the day with our CRISPR Goddess.

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 for FSHD.

Apr 12, 202301:32:27
The return of Brad (the angry dad).

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.

Mar 26, 202301:51:18
Rare Disease Day and Accountability

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, myostatin inhibition, and cell therapy trials.

Mar 10, 202301:40:40
Reintroducing the MyFSHD podcast

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 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.

Feb 25, 202301:29:49
"A" is for Avidity

"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 conjugated siRNA designed specifically to knockdown the DUX4 mRNA in FSHD.

Feb 13, 202301:13:58
We have a lot in common with pigs. "Don't flatter yourselves", reply the pigs :).

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

"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!

Feb 06, 202301:27:34
Our "State of the Field" address.

Our "State of the Field" address.

"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.

Jan 27, 202301:47:15
Happy 2023 from MyFSHD!

Happy 2023 from MyFSHD!

"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.

Jan 15, 202301:32:04
Merry Christmas, Happy Hanukkah, and Happy Holidays from MyFSHD

Merry Christmas, Happy Hanukkah, and Happy Holidays from MyFSHD

"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.

Dec 25, 202201:45:42
Antisense, gene therapy, and stem cell news, and the Blues Brothers

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

"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.

Dec 15, 202201:26:04
The best humanized FSHD muscle xenograft mouse model with Dr. Bob Bloch.

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

"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.

Dec 02, 202201:54:16
We have a lot to be thankful for!

We have a lot to be thankful for!

"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.

Nov 24, 202201:56:55
More on FSHD research testing, CRISPR, and minipigs

More on FSHD research testing, CRISPR, and minipigs

“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.

Nov 20, 202201:37:56
Catch up on some news of the week then sit down with FSHD researcher Maryam Farooqi

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

"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).

Nov 10, 202201:37:34
News of the week on Fulcrum and CRISPR gene therapy.

News of the week on Fulcrum and CRISPR gene therapy.

"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.

Nov 06, 202201:13:36
Happy Nevada Day! Today we talk about early onset FSHD and getting adolescents into clinical trials.

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

"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.

Oct 31, 202201:45:40
Monopoly money in science and to test, or not to test, (your kids) for FSHD

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

"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.

Oct 21, 202201:35:43
Keeping up with even more investment and more technology coming into the FSHD space.

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

"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.

Oct 13, 202201:14:10
More clinical trial discussion with our FSHD Mom and Dad.

More clinical trial discussion with our FSHD Mom and Dad.

"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.

Oct 05, 202201:49:40
Update on clinical trials for FSHD

Update on clinical trials for FSHD

"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.

Oct 01, 202201:13:48
Catching up on FSHD, Saturday Sept 24th, 2022

Catching up on FSHD, Saturday Sept 24th, 2022

“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.

Sep 24, 202201:25:26
Live from Sydney, Australia, it's MyFSHD!

Live from Sydney, Australia, it's MyFSHD!

"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.

Sep 17, 202201:23:54
Brunch with Maryam (and Takako and Peter). Our 50th episode!

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

"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.

Sep 11, 202201:29:28
FSHD catch-up, September 7, 2022

FSHD catch-up, September 7, 2022

"The secret's in the sauce."  We discuss some recent news in the neuromuscular disease space and FSHD publications on cell therapy and biomarkers.

Sep 08, 202201:03:45
More CRISPR questions from the audience.

More CRISPR questions from the audience.

"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.

Sep 01, 202201:19:58
Kari Cilliers, a medical student from South Africa found her way to Nevada to learn about FSHD

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

"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.

Aug 27, 202201:09:58
Our FSHD Dad is back with more questions, comments and concerns on biomarkers, clinical trials, MRI, and supplements

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

"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!

Aug 19, 202201:49:51
Fixing FSHD down under (and everywhere) with FSHD Global Research Foundation

Fixing FSHD down under (and everywhere) with FSHD Global Research Foundation

“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.

Aug 13, 202201:34:38
More on nutrition, supplements, and lifestyle/exercise.

More on nutrition, supplements, and lifestyle/exercise.

"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.

Aug 11, 202201:48:28
Saturday catchup and a little bit more on FSHD-like minipigs, Aug 6, 2022

Saturday catchup and a little bit more on FSHD-like minipigs, Aug 6, 2022

"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.

Aug 06, 202201:07:41
Bringing home the bacon with Jenny, Ben, Peter, and FSHD-like minipigs.

Bringing home the bacon with Jenny, Ben, Peter, and FSHD-like minipigs.

"Go the distance."  Jenny Hasenjaeger of MyFSHD sits down with Ben Brittain to discuss his FSHD and what he intends to do about it.

Aug 03, 202201:56:15
Weekend catchup, July 31, 2022; hanging out in the high Sierra Nevada for summer Sunday brunch with Brad and Peter discussing all things FSHD.

Weekend catchup, July 31, 2022; hanging out in the high Sierra Nevada for summer Sunday brunch with Brad and Peter discussing all things FSHD.

"We'll burn that bridge when we get to it."  Our FSHD dad lets rip with his thoughts and opinions on all things FSHD in a whirlwind discussion with Dr. Jones trying to keep up.  And bears; a little bit on bears.

Jul 31, 202201:38:25
We ain't dead yet, so we can get better. Welcome to the 40th podcast episode!

We ain't dead yet, so we can get better. Welcome to the 40th podcast episode!

"So shines a good deed in a weary world."  It is our 40th episode, so we are recapping and reintroducing ourselves to the wider audience.

Jul 27, 202201:09:32
Saturday FSHD catch-up, July 16th 2022

Saturday FSHD catch-up, July 16th 2022

"What am I working on? Uhhh, I'm working on something that will change the world and human life as we know it."  It's not teleportation, but, at your request, Drs Charis Himeda and Peter Jones discuss the recent announcement of Epic Bio's entry into the FSHD therapeutic space.

Jul 26, 202201:17:44
MyFSHD welcomes Chris Carrino

MyFSHD welcomes Chris Carrino

"Let’s win this one for all the small schools that never had a chance to get here."  Everything is possible with talented people sincerely dedicated to a cause.  Today MyFSHD welcomes one of the best, professional sports broadcaster Chris Carrino, founder of the Chris Carrino Foundation for FSHD and an FSHDer himself, to discuss his foundation, funding research, and his journey with FSHD.

Jul 20, 202201:38:19
FSHD in the UK with Kate and Dr. Channa Hewamadduma

FSHD in the UK with Kate and Dr. Channa Hewamadduma

"I've never had anything you doctors didn't try to cure with leeches."  Kate Fowles hosts Dr. Channa Hewamadduma, Consulting Neurologist at the University of Sheffield Neuroscience Institute, as they discuss FSHD and clinical trial readiness.

Jul 13, 202201:33:27
A little bit deeper into DNA methylation.

A little bit deeper into DNA methylation.

"You stay classy, San Diego. I'm Ron Burgundy?"... "Dammit, who typed a question mark on the TelePrompter?... Anything you type, Burgundy will read!" Dr. Peter Jones talks recent developments in the FSHD space, DNA methylation, and answers questions on diagnostics and triggers of pathology.

Jul 09, 202201:09:57
Back in Reno, Dr. Peter Jones fields some questions from all of you.

Back in Reno, Dr. Peter Jones fields some questions from all of you.

"Here they are, back after their exclusive three-year tour of Europe, Scandinavia and the subcontinent."  MyFSHD is back in Reno, but the work never stopped, and we are answering some common questions today.

Jul 01, 202252:42
Saturday catchup with questions and comments around FSHD, June 25, 2022

Saturday catchup with questions and comments around FSHD, June 25, 2022

"It's a bold strategy, Cotton. Let's see if it pays off for 'em."  Coming to you from NYC where Dr. Peter Jones is with Ms. Nguyen Cam Thi talking about her personal journey with FSHD and the impact of a trip to the US and meeting with other patients, and we answer some listener questions on FSHD.

Jun 25, 202201:07:27