#musculardystrophy — Public Fediverse posts
Live and recent posts from across the Fediverse tagged #musculardystrophy, aggregated by home.social.
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RE: https://todon.eu/@regendans/116579546000043721
Everyone please read this! This is horrid! We can't let people be treated like this ! A #MustReadArticle
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RE: https://todon.eu/@regendans/116579546000043721
Everyone please read this! This is horrid! We can't let people be treated like this ! A #MustReadArticle
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The final episode of Cycle The Nations World Record adventure is now on YouTube, and you can own can own a part of the adventure. The charity auction for Cyclus Minor is now live here: https://ebay.us/m/yjV7t1 He is looking a bit worse for wear, but that's all a part of his story. All proceeds are going to Muscular Dystrophy UK, own a part of history, and help out a good cause!
#Warhammer #Warhammer40K #WarhammerCommunity #MuscularDystrophy #Charity
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The final episode of Cycle The Nations World Record adventure is now on YouTube, and you can own can own a part of the adventure. The charity auction for Cyclus Minor is now live here: https://ebay.us/m/yjV7t1 He is looking a bit worse for wear, but that's all a part of his story. All proceeds are going to Muscular Dystrophy UK, own a part of history, and help out a good cause!
#Warhammer #Warhammer40K #WarhammerCommunity #MuscularDystrophy #Charity
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Preschool Teacher Spots Symptoms and Tells Parents, Leading to Child’s Early Diagnosis With Rare Disease
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https://youtu.be/i3u_2MaqgoI?si=vnPM1Lx6mBcvYcSs New video is on YouTube. This week I am painting a Space Marine which will be taking part in a World Record breaking challenge to cycle to every county in the UK raising money for Muscular Dystrophy UK.
#Warhammer #Warhammer40K #Warhammer40000 #PaintingWarhammer #WarhammerCommunity #PaintingMiniatures #PaintingMinis #GamesWorkshop #BoardGames #YouTube #MDUK #MuscularDystrophy #Nerdlings
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https://youtu.be/i3u_2MaqgoI?si=vnPM1Lx6mBcvYcSs New video is on YouTube. This week I am painting a Space Marine which will be taking part in a World Record breaking challenge to cycle to every county in the UK raising money for Muscular Dystrophy UK.
#Warhammer #Warhammer40K #Warhammer40000 #PaintingWarhammer #WarhammerCommunity #PaintingMiniatures #PaintingMinis #GamesWorkshop #BoardGames #YouTube #MDUK #MuscularDystrophy #Nerdlings
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Hey, folks?
Mike Ahlers is an old friend and colleague from way back when. He's a semi-pro bicycle racer these days. He's riding in the 2025 Triple Bypass (https://www.triplebypass.org/) in Colorado to raise money for #MuscularDystrophy research, by way of the #FSHD Society (https://www.fshdsociety.org/about-us/).
Both Mike and his sister have #MuscularDystrophy (specifically, facioscapulohumeral muscular dystrophy), so he's got skin in the game.
If you've got a few bucks to spare would you please consider donating to his ride?
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"We are...convinced that this has opened the door to the development of new therapeutic approaches for the treatment of Duchenne muscular dystrophy. In particular, this could be the case for patients with mutations that have so far been difficult to address," said Dr Didier Stainier, Director of the Max Planck Institute for Heart and Lung Research, among other appointments.
#MuscularDystrophy #DMD #health #medicine #genetics #research #science
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"We are...convinced that this has opened the door to the development of new therapeutic approaches for the treatment of Duchenne muscular dystrophy. In particular, this could be the case for patients with mutations that have so far been difficult to address," said Dr Didier Stainier, Director of the Max Planck Institute for Heart and Lung Research, among other appointments.
#MuscularDystrophy #DMD #health #medicine #genetics #research #science
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Very disappointing news on the Duchenne muscular dystrophy front. Elevidys, the only available gene therapy for DMD has shown no significant improvement in motor function in kids receiving the treatment after 1 year. However, certain secondary outcomes showed some benefit.
It remains to be seen if FDA will allow it to stay on the market in the US, given that there are no other available treatments for DMD.
https://www.ajmc.com/view/sarepta-joins-pfizer-in-dmd-gene-therapy-trial-failures
#Duchenne #musculardystrophy #raredisease #genetherapy #elevidys #biotech #pharma
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Scientists create a new approach for muscular dystrophy gene therapy. The researchers are hooing to start human clinical trials within about two years.
#MuscularDystrophy #DMD #genetics #health #medicine #biology #biotechnology
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Scientists create a new approach for muscular dystrophy gene therapy. The researchers are hooing to start human clinical trials within about two years.
#MuscularDystrophy #DMD #genetics #health #medicine #biology #biotechnology
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#DUX4 modulates translational regulators to suppress protein synthesis while promoting expression of DUX4-induced genes, suggesting a role in cell state conversion during development (and pathological role in #cancer or #MuscularDystrophy) #PLOSBiology https://plos.io/48FLJAe
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#DUX4 modulates translational regulators to suppress protein synthesis while promoting expression of DUX4-induced genes, suggesting a role in cell state conversion during development (and pathological role in #cancer or #MuscularDystrophy) #PLOSBiology https://plos.io/48FLJAe
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"It felt like the Hunger Games":
Between 6th birthdays that came too soon & insurance companies denying coverage, families scramble to get their 5 year-olds treated under Sarepta's narrow #FDA approval for #DMD
What a gut punch ending.
#health #healthcare #biotech #genetherapy #insurance #duchenne #musculardystrophy #parenting #medicine
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"It felt like the Hunger Games":
Between 6th birthdays that came too soon & insurance companies denying coverage, families scramble to get their 5 year-olds treated under Sarepta's narrow #FDA approval for #DMD
What a gut punch ending.
#health #healthcare #biotech #genetherapy #insurance #duchenne #musculardystrophy #parenting #medicine
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Man with muscle-wasting condition completes gruelling Ironman triathlon | indy100
https://www.indy100.com/news/man-with-muscle-wasting-condition-completes-gruelling-ironman-triathlon#Ironman
#IronmanTriathlon
#FaciocapulohumeralMuscularDystrophy
#MuscularDystrophy
#MD
#IronManAustralia -
Gene therapy with AAV vectored micro-dystrophin for Duchenne muscular dystrophy. My latest #ClinicalPipeline column for #NatureMedicine.
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Gene therapy with AAV vectored micro-dystrophin for Duchenne muscular dystrophy. My latest #ClinicalPipeline column for #NatureMedicine.
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Prepare for any life challenges with a nice ice bath! A mindfulness and meditation event on Monday 1 May near Huddersfield will launch a new charity - 'Hero’s Fight Against DMD' (Duchenne muscular dystrophy) | Rachel Devereux
#DMD #musculardystrophy #Huddersfield #HerosFight
https://yorkshirebylines.co.uk/news/health/mindfulness-and-meditation-event-to-launch-new-charity-for-boy-with-dmd/ -
Prepare for any life challenges with a nice ice bath! A mindfulness and meditation event on Monday 1 May near Huddersfield will launch a new charity - 'Hero’s Fight Against DMD' (Duchenne muscular dystrophy) | Rachel Devereux
#DMD #musculardystrophy #Huddersfield #HerosFight
https://yorkshirebylines.co.uk/news/health/mindfulness-and-meditation-event-to-launch-new-charity-for-boy-with-dmd/ -
#ElonMusk attacks disabled employee who sold his company to #Twitter, which included a management job in the contract, in 2021, before Elon.
The man is disgusting. Taking jabs at his muscular dystrophy.
Thread at #Nitter from the employee who shares that tweet. It's a moving thread Shared it via Nitter so you won't be going to Twitter.
https://nitter.net/iamharaldur/status/1633082707835080705#m -
#ElonMusk attacks disabled employee who sold his company to #Twitter, which included a management job in the contract, in 2021, before Elon.
The man is disgusting. Taking jabs at his muscular dystrophy.
Thread at #Nitter from the employee who shares that tweet. It's a moving thread Shared it via Nitter so you won't be going to Twitter.
https://nitter.net/iamharaldur/status/1633082707835080705#m -
#StemCells and #GeneTechnology against local #muscledefects, acute muscle wasting disorders, genetic #muscularDystrophy:
Start-up #MyoPax is breaking new ground to fight #RareDiseases.#RareDiseaseDay @rarediseaseday @campusbuch
https://www.mdc-berlin.de/news/news/deep-expertise-and-bold-vision-myopax-launches-berlin -
#StemCells and #GeneTechnology against local #muscledefects, acute muscle wasting disorders, genetic #muscularDystrophy:
Start-up #MyoPax is breaking new ground to fight #RareDiseases.#RareDiseaseDay @rarediseaseday @campusbuch
https://www.mdc-berlin.de/news/news/deep-expertise-and-bold-vision-myopax-launches-berlin -
If you didn’t need to do much to support research into #LongCovid, #MECFS, #MuscularDystrophy, and other #PostViralSyndromes, would you?
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This is one of those stories that either is the start of something huge or will sink like a stone: not much in between.
#Tessera’s claims are ambitious but not unprecedented. One of our clients does something similar with stem cells, and they have a product that’s actually made it to market. As far as I can tell, Tessera is claiming the ability to do this in all kinds of cells, with whole living organisms. Which would be a really big deal.
From a bioinformatics / biostatistics perspective (you knew that was coming) I have Questions about consistency. Doing it once, or ten times, or a thousand, isn’t enough. You need to do it trillions, very accurately indeed. With cell cultures you just screen out the cells that didn’t do what you wanted and keep the ones that did: insert “bury their mistakes” joke here. And with stem cells it’s particularly easy to know when you have it right.
“65 percent efficiency—20 times the rate at which it performed unintended or errant edits” sounds quite good on a cell-by-cell basis, although I’d like to see the error bars on those numbers. In a patient, it would be an unmitigated disaster. Congratulations, we’ve cured your muscular dystrophy … and given you ten different types of cancer.
More generally, their secrecy does not fill me with confidence, of the 95% variety or any other. As I’ve said many times before, science done in secret isn’t science at all, it’s alchemy.
Well. It’s early days yet. Let’s just hope Tessera isn’t another Theranos, because I am really tired of that. If they do achieve any of what they’re claiming, I’ll be cheering them on.
#genetics #geneediting #genewriting #stemcell #bioinformatics #musculardystrophy #cancer
https://www.the-scientist.com/bio-business/can-gene-writing-deliver-what-gene-editing-can-t-70740
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Finally, an #introduction.
My name is Tim, I’m 49 years old, and I have FSH #MuscularDystrophy. I live in a neurological centre in the UK, and I write about living with a #disability (link in my bio).
I was a programmer when I worked (dBase and #FoxPro), and still tinker for fun, mostly in #PHP.
I like #ScienceFiction and #ScienceFact , especially #astronomy / space-based stuff, and consume related movies and books. I have music on at every possible moment in my day.
