"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.

https://www.statnews.com/2023/08/16/duchenne-muscular-dystrophy-sarepta-elevidys-gene-therapy-eligible-boys/

#health #healthcare #biotech #genetherapy #insurance #duchenne #musculardystrophy #parenting #medicine

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

First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids | AP News

>The Food and Drug Administration approval provides a new option for some patients with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes weakness, loss of mobility and early death. It almost always affects males.
https://apnews.com/article/fda-gene-therapy-duchenne-muscular-dystrophy-approval-82a82cfb67ef9fb6cf75cd3b5865c138 #musculardystrophy

Gene therapy with AAV vectored micro-dystrophin for Duchenne muscular dystrophy. My latest #ClinicalPipeline column for #NatureMedicine.

#drugdevelopment #SciComm #MuscularDystrophy #Pharma

https://www.nature.com/articles/d41591-023-00052-4

Referenced link: https://medicalxpress.com/news/2023-04-bold-therapy-delivery-method-treatment.html
Discuss on https://discu.eu/q/https://medicalxpress.com/news/2023-04-bold-therapy-delivery-method-treatment.html

Originally posted by Phys.org / @physorg_com: http://nitter.platypush.tech/medical_xpress/status/1651957162577362949#m

RT by @physorg_com: Bold new therapy delivery method shows initial promise as treatment for Duchenne #musculardystrophy @CellCellPress https://linkinghub.elsevier.com/retrieve/pii/S0092867423003306 https://medicalxpress.com/news/2023-04-bold-therapy-delivery-method-treatment.html

Referenced link: https://medicalxpress.com/news/2023-04-protein-bves-important-role-muscular.html
Discuss on https://discu.eu/q/https://medicalxpress.com/news/2023-04-protein-bves-important-role-muscular.html

Originally posted by Phys.org / @physorg_com: http://nitter.platypush.tech/medical_xpress/status/1643699914784878592#m

RT by @physorg_com: Researchers investigate the protein BVES and its important role in #musculardystrophy @iumedschool @NatureComms https://www.nature.com/articles/s41467-023-37496-8 https://medicalxpress.com/news/2023-04-protein-bves-important-role-muscular.html

One would think #ElonMusk, who is disabled himself with #autism and malignant #Narcissist personality disorder, wouldn’t make fun of a fellow disabled laid off wheelchair-bound #Twitter employee with #MuscularDystrophy!

“Musk implied that #Haraldur, known as Halli, had not been fulfilling his job requirements at Twitter, while Halli asked whether Twitter would pay him the severance the company owes him now that he has been let go.”

https://www.icelandreview.com/business/elon-musk-and-halli-spar-over-termination-at-twitter/

One would think #ElonMusk, who is disabled himself with #autism and malignant #Narcissist personality disorder, wouldn’t make fun of a fellow disabled laid off wheelchair-bound #Twitter employee with #MuscularDystrophy!

“Musk implied that #Haraldur, known as Halli, had not been fulfilling his job requirements at Twitter, while Halli asked whether Twitter would pay him the severance the company owes him now that he has been let go.”

https://bit.ly/3ZMi4A6

Elon Musk 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.
https://nitter.net/iamharaldur/status/1633082707835080705#m

#disability #MuscularDystrophy

http://www.haraldurthorleifsson.com/ ❤️❤️🙏🏻🕊✌🏻🌻🌎🌍🌏🎉 great #branding ❣️ #Shaws has a #MuscularDystrophy foundation I think? MDA - right now as well when you check out donate option. ❤️🙏🏻🕊✌🏻🌻

#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

Referenced link: https://medicalxpress.com/news/2023-02-toxic-protein-linked-muscular-dystrophy.html
Discuss on https://discu.eu/q/https://medicalxpress.com/news/2023-02-toxic-protein-linked-muscular-dystrophy.html

Originally posted by Phys.org / @physorg_com: http://nitter.platypush.tech/medical_xpress/status/1626657742734647317#m

RT by @physorg_com: Toxic protein linked to #musculardystrophy and arhinia @NIHforHealth @ScienceAdvances https://www.science.org/doi/10.1126/sciadv.abq7744 https://medicalxpress.com/news/2023-02-toxic-protein-linked-muscular-dystrophy.html

The main difficulties I may encounter are financial, physical and psychological problems but I hope this year will be the year of change for me and all my friends in the chronic illness community.
#MuscularDystrophy
#LGMD
#chronicillness
#DisabilityTwitter

To sum up ,I abandon my high hope to deal with the fact that control my movement and will be back to dream big again as soon as I feel it's possible to reach the dreams I long for.
#MuscularDystrophy
#LGMD
#chronicillness
#DisabilityTwitter

For the time being, I see it's better to think in building my own life to suit my current situation.
#LGMD
#MuscularDystrophy
#chronicillness
#DisabilityTwitter

Also it sounds a cazy idea to think in travelling to an other country where I can find the healthcare I need , subsequently the improvement I always dream of , though it's valid in my opinion.
#LGMD
#MuscularDystrophy
#chronicillness
#DisabilityTwitter

For instance ,I hoped that I might be able to know the subtype of LGMD I have by a way or an other as it was not and is still impossible to know it here.
#LGMD
#MuscularDystrophy
#chronicillness
#DisabilityTwitter

I raised my hope so high at the beginning that I might find a fundamental solution for Muscular Dystrophy I had, that's to say,LGMD ,but finally,I realized it almost impossible to find that solution at least currently.
#LGMD
#MuscularDystrophy
#chronicillness
#DisabilityTwitter

I think it won't be much terrifying,if the flesh isn't lost progressively .
#LGMD
#MuscularDystrophy

More and more people are discovering that the Muscular Dystrophy Association is no longer supporting those it claims to support, and they want to do something about it. My latest in Medium.

https://despinakarras.medium.com/abandoned-by-the-muscular-dystrophy-association-45906dc83976

#nonprofit #mismanagement #mda #musculardystrophy