pTDP-43 levels correlate with cell type–specific molecular alterations in the prefrontal cortex of C9orf72 ALS/FTD patients | PNAS pnas.org/doi/10.1073/... Interesting to see microglia changes are seen first then followed by oligodendrocytes and astrocytes - suggests cascade

Rethinking phase 2 trials in amyotrophic lateral sclerosis academic.oup.com/brain/articl... Well-thought out discussion - basically if you want to run 6-month Phase 2 trials - use biomarkers for your go/no-go decision. If you want to use ALSFRS-R - you need a longer trial

Read our new perspective piece published in @mja.com.au. We explore the role of GCs in genomic medicine and current professional challenges. What started as passionate conference discussions has turned into a great perspective piece! To read the paper: onlinelibrary.wiley.com/doi/10.5694/...

Characteristic symptoms of MND/ALS are preceded by sleep disturbances Findings suggest that sleep disorders appear before the onset of motor impairment and respiratory issues healthcare-in-europe.com/en/news/aml-... www.science.org/doi/10.1126/...

A lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection Better alternative to intrathecal (spinal) injections Potential for MND gene therapies? Only tested in mice so far... www.nature.com/articles/s41... www.news-medical.net/news/2025021...

Neurological Alliance Australia (NAA) (MND Australia area member) are advocating for the creation of a Taskforce for Neurological Conditions to urgently address six critical areas of need. It takes just a few minutes to send an email to your local MP countusin.good.do/naa/count-us...

Not specifically MND but the concussion/brain injury association with neurodegeneration is still not finalised Sports-related concussion not associated with long-term cognitive or behavioural deficits: the PROTECT-TBI study jnnp.bmj.com/content/earl...

Genetic testing for #ALS #MND should go beyond identifying a single mutation. Even if a known genetic cause is found. A comprehensive analysis provides a clearer understanding of the condition and can help guide care &research efforts. Happy to help in this fantastic project.

Some challenging ideas - a very well thought out hypothesis; Amyotrophic lateral sclerosis represents corticomotoneuronal system failure - Eisen - Muscle & Nerve - Wiley Online Library onlinelibrary.wiley.com/doi/10.1002/...

Pridopidine in ALS - HEALEY Platform Trial Outcome "In this 24-week study, pridopidine did not impact the progression of ALS" Is 6-months enough to see change? Healey now moving to 9-month trials - hopefully that will produce more definitive outcomes jamanetwork.com/journals/jam...

CNM-Au8 in ALS - HEALEY Platform Trial outcomes No significant positive effects. Government funded EAP underway. Previous trials showed efficacy in longer-term follow-up - Healey only 6-months Larger Phase 3 trial under consideration and Clene in discussions with FDA jamanetwork.com/journals/jam...

This morning, the National Science Foundation fired 168 probationary employees, effective immediately. The number represents roughly 10% of its workforce. Follow Science’s coverage of President Donald Trump’s impact on U.S. research and science globally. ⬇️ scim.ag/40XtSSi

We're aware there is still debate going on, on the merits of ellorarxine. We've made our position on this clear, which is that from the data published so far, it is one of many drugs or compounds in a similar position. We'll be watching developments closely, as we do with all such situations.

We are alarmed by proposed funding cuts that would devastate the fight against ALS. Slashing funding for NIH will hinder efforts to turn ALS from fatal to livable and cure it. Congress MUST reject these cuts! We need your voice NOW more than ever. bit.ly/NIH-funding-...

How does EXPERTS-ALS fit into the MND global drug development process? #als #mnd united2endmnd.org/2025/02/10/e... #united2endmnd

Our study on the safety and tolerability of Trimetazidine in #ALS @braincomms.bsky.social Trimetazidine reduced oxidative stress markers and energy expenditure, warranting a follow up study @rpavaneijk.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social academic.oup.com/braincomms/a...

“These are real consequences, longer waits for cures and for diagnosis, slower scientific progress, losing out to competitors… and fewer jobs. Those who are facing any health challenges will suffer from less biomedical research.” — D. Skorton Via @nytimes: www.nytimes.com/2025/02/07/u...

The NIH’s drastic cut to indirect cost rates is a critical threat to U.S. research infrastructure www.statnews.com/2025/02/08/n... via @statnews.com

Can't do your research in the dark or in the garden - infrastructure funding is vital and much of that comes through indirect costs!

Good to see this Australian-led study ready for the next phase; Open-label phase 2a study to assess the safety and tolerability of trimetazidine in patients with ALS academic.oup.com/braincomms/a... @rpavaneijk.bsky.social @ngoshyuan.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social

With Tofersen having the potentially to significantly slow disease - would such treatments as this potentially return some function if used in conjunction (sorry behind paywall) First-in-human study of epidural spinal cord stimulation in individuals with SMA doi.org/10.1038/s415...

Only 7 patients but encouraging finding Currently under review for approval by TGA in Australia Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real‐world” setting onlinelibrary.wiley.com/doi/10.1002/...

We are aware of recent discussions within the MND community regarding research into ellorarxine. Read our update here 👇 www.myname5doddie.co.uk/whats-on/art...

Plenty of caveats but a decent number of participants and to my non-expert eye a reasonable analysis; Longer disease progression milestone-free time in people with ALS treated vs not treated with iv edaravone: results from an administrative claims analysis becarispublishing.com/doi/10.57264...

Very useful study - would be great to see further studies adding to the data and develop consensus numbers. Full article: Estimating the minimum important difference in the ALSFRS-R-instrument in people living with MND www.tandfonline.com/doi/full/10....

Muscle ultrasound aids diagnosis in amyotrophic lateral sclerosis Andrew Hannaford, Nathan Pavey, Parvathi Menon, Mehdi A J van den Bos, Matthew C Kiernan, Neil Simon, Steve Vucic. www.sciencedirect.com/science/arti...

A new collaborative paper is out... a surprising new non-conventional phenotype in ALS, and a way to treat it: www.science.org/doi/10.1126/...

Early-onset sleep alterations found in patients with amyotrophic lateral sclerosis are ameliorated by orexin antagonist in mouse models | Science Translational Medicine www.science.org/doi/10.1126/...

Neale Daniher, a campaigner in the fight against motor neurone disease and a former champion Essendon footballer, is the 2025 Australian of the Year,

"a federally funded, ALS-specific, end-to-end clinical trials network would be hugely beneficial for the ALS field and could substantially increase the number of major trials in ALS, accelerate drug discovery, and bring about much needed research infrastructure support and efficiencies"

Likely similar in Australia - our forthcoming Impact Report will provide updated data on this topic Full article: Amyotrophic lateral sclerosis estimated prevalence cases from 2022 to 2030, data from the national ALS Registry www.tandfonline.com/doi/full/10....

Critical to keep these cowboy organisations away from our community Stem Cell Institute Co-Founders and Companies Banned from Marketing Stem Cell Treatments and Ordered to Pay More Than $5.1 Million for Refunds and Civil Penalties www.ftc.gov/news-events/...

Interesting study but definitely needs a follow-up to show how no change in progression rate can still have an impact on survival? Transcranial static magnetic stimulation for amyotrophic lateral sclerosis: phase 2 trial pubmed.ncbi.nlm.nih.gov/39185360/

A great organisation and now have Australian-friendly meeting times!

We need MORE not LESS regulatory oversight of the marketing of unproven therapies. Nice to see this action...👇 Stem Cell Institute Co-Founders & Companies Banned from Marketing Stem Cell Treatments and Ordered to Pay More Than $5.1 Million for Refunds & Civil Penalties www.ftc.gov/news-events/...

Good overview of neurofilaments as potential biomarkers 'Blood level of neurofilament light chain as a biomarker for neurological disorders' bit.ly/4gR6hJE

🚨 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 🔗 ow.ly/hfVf50UFSge 🛡️ Be protected from misinformation. From tandfonline.com

We're hiring Communications and Engagement Managers These roles will manage funded projects to support people living with MND Join a fantastic team t.co/gSIn3EOBYH