EndALS

Momentum is building. Let’s #EndALS. Not an organization or charity. Just a Twitter account.

[ALS Advocacy @alsadvocacy]

Such duplication of work in ALS Land. Such mixed messages.
If u can only participate in 1 natural history study, please make it ALL ALS. It is well-designed & well-funded. NIH really stepped up. To get the mass of data in it will make a difference. https://t.co/6edG2HKGvv

[EndALS @FinishALS]

Insurance companies are evil. “Clinical benefit has not been established”….that’s literally why they performed a phase 3 study.

[STAT @statnews]

Shelby Kinsey lives with a rare, fast-progressing form of ALS — but she has to spend her precious time fighting for insurance coverage of a drug that helps. https://t.co/NSJGi7GCvE

[ALS/MND Alliance @ALSMNDAlliance]

🚨 Warning: Websites and social media are full of claims with high promises for ALS/MND treatments. Read about ten red flags you should watch for from #ALSUntangled 🔗 https://t.co/rpFXIB87PT 🛡️ Be protected from misinformation.

[Shyuan Ngo 🧠🔬🧫👩🏻‍🍳😻 @NgoShyuan]

Our study on the safety and tolerability of Trimetazidine in #ALS @braincomms
Trimetazidine reduced oxidative stress markers and energy expenditure, warranting a follow up study.
@rpavaneijk @Fred_Steyn @AmmarAlChalabi

https://t.co/Fmz4Vq90AG

[Sri Kosuri @srikosuri]

It’s been a tough few weeks. My 10yo daughter was diagnosed with a very rare, aggressive cancer called interdigitating dendritic cell sarcoma (IDCS). I’m reaching out to identify clinicians/patients who have encountered pediatric IDCS, indeterminate dendritic cell histiocytosis or other (non-LCH) histiocytic sarcomas cases.

I'm trying to understand non-surgical chemo and targeted therapy options, new pathology markers to better diagnose subtypes/treatments, and any data on progression in pediatric patients. Please feel free to share – I’m trying to cast a wide net due to the rarity of this condition and how little is known.

People can contact me directly at my first name (as written in my profile) at https://t.co/ubo0zQRMn0.

[Adam Feuerstein ✡️ @adamfeuerstein]

Some sad news to report:

Austin Leclaire was a remarkable young man with Duchenne muscular dystrophy who raised awareness about his devastating disease, and as a patient advocate, played a pivotal role in spurring the development of the first medicines to treat it. Austin died on Feb. 1, according to an announcement from his family. He was 26.

I didn’t know Austin well, but I first met him in 2012 at an event in Boston organized by his mother, Jennifer McNary, and other Duchenne advocates who were pushing Sarepta Therapeutics to speed up the development of eteplirsen, its first drug to treat Duchenne. I can’t recall any of the speeches made that night, but I have a vivid memory of Austin, then already in a wheelchair, and his younger brother Max, also born with Duchenne and bouncing around the room like a jitterbug, captivating the crowd.

Eteplirsen was a controversial drug, even back then. But what those gathered that night hoped for — as they watched two brothers playing joyfully, waiting impatiently for the adults to stop talking — were new treatments that could end a terrible disease. The event concluded with McNary and the other advocates presenting Sarepta's then-CEO Chris Garabedian with an hourglass, because in their words, time was something boys with Duchenne couldn’t afford to lose.

Austin was a champion for the Duchenne community. His advocacy work, his optimism, his self-described “raunchy comedy act” and participation in competitive sports — it all inspired people. He was deeply loved by his mom, who was so proud of the man he had become.

“My wish for his legacy is what he would have wanted — for people to remember him loudly. He loved the spotlight and aspired to change the world,” McNary told me via email. “He was an amazing son, big brother, friend and much more. I want us all to remember his contributions to the world because he had every reason to wallow and be selfish but he never complained and did everything he could to serve.”

My condolences to Jenn, Max, and their extended family at this difficult time. Austin had a huge impact on everyone he met and he will be remembered fondly.

[Rothstein Lab @Rothstein_Lab]

Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real-world” setting
https://t.co/LN9plC925b

[ALS News Today @alsnewstoday]

Treatment with the therapy was also associated with a roughly 50% slowing of respiratory declines. Review all findings here: https://t.co/tBepzV90jL

#ALS #AmyotrophicLateralSclerosis #ALSDisease #ALSTreatment #ALSResearch

[ALS Advocacy @alsadvocacy]

New immune cell therapy benefits lab models of ALS and shows positive results in an individual with the disease https://t.co/hiimNp455s via @medical_xpress

[EndALS @FinishALS]

@Jeanc9orf72 @rpavaneijk @BlockAntonious @alsadvocacy I don’t think so. Still have a equal sample size in the group.

[EndALS @FinishALS]

@Jeanc9orf72 @rpavaneijk @BlockAntonious @alsadvocacy I don’t have a good answer. I’m not designing trials and I’m not a researcher. I have an understanding of trial design from a masters obtained in a much less complicated field and don’t have the qualifications to make decisions that these researchers do. Just want better for all.

[EndALS @FinishALS]

@Jeanc9orf72 @rpavaneijk @BlockAntonious @alsadvocacy This stuff happens. Is it right? No, but you can understand where they’re coming from. That sullies the data already. That keeps trials from filling. All because of fear of just being given nothing and having people watch you decline.

[EndALS @FinishALS]

@Jeanc9orf72 @rpavaneijk @BlockAntonious @alsadvocacy “More likely” meaning still the element of uncertainty regarding randomization. Are we getting worse data from the pooled placebo from the platform trial?

[EndALS @FinishALS]

@Jeanc9orf72 @rpavaneijk @BlockAntonious @alsadvocacy I do understand what you’re saying, however. By definition it is not an act of harm.

[EndALS @FinishALS]

@Jeanc9orf72 @rpavaneijk @BlockAntonious @alsadvocacy Is their life not as important as future patients? Should we ask them to sacrifice several months if they’re on placebo just so others down the line can preserve function. Fail to see how hybrid model dilutes the data so badly that it can’t work.

[EndALS @FinishALS]

@rpavaneijk @BlockAntonious @alsadvocacy Hybrid does seem to get around this suboptimal approach, does it not? You can still have a (much smaller) placebo group which keeps the blinding in place as participants and investigator won’t know who is getting the real deal - only that the chance is decreased they aren’t.

[EndALS @FinishALS]

Searching for something on X and the name that appears on the toolbar is someone that lost their ALS fight several years ago and I had to stop for a second. We only conversed on Twitter, but I could tell he was a great man. This disease just sucks.

[Raffaele Di Giacomo, PhD @sciqst]

The study showcases a critical link between stress-induced TDP-43 nuclear condensation and its downstream effects on splicing and STMN2 levels. This underscores the molecular intricacies behind neurodegenerative processes, potentially highlighting novel therapeutic targets. For more in-depth biomedical insights, explore https://t.co/4Y9Imt8uIJ, an AI-driven platform to generate comprehensive biomedical reviews. #Medicine #Neuroscience #OpenAI Sciqst powered by OpenAI.