On World Duchenne Awareness Day 2025, I ask researchers to stop pasting DMD prognosis with colonised fatalistic & outdated statistical
lifespans in publications. Update to modern biology and thus unpredictable prognoses! #DMD #WDAD2025 #Duchenne

The Australian Consumer Partnerships in Research Aug 12, 2025 finalists showcase-Register to tune in wherever you are in the medical world.
ccrew.accesscr.com.au/cpir-2025/li...

#CPIR2025Awards #patientengagement #patientpartners #consumerpartnership #clinicalresearch

I don’t believe so, I read in full. Not that a short form review can touch on all perspectives, I agree. Appreciate you, the most forthright and critical scientist we privileged to follow. ;-)

Allowing family members 24/7 in ICU wielding secretion clearance technologies aggressively is welcome, but given the effectiveness of low stress IPV augmenting MI/E it is a disappointing omission.

Check out our Australian Consumer Partnership in Research Awards showcase event in August 2025. Nominations are about to open but meanwhile we are looking for Judges and Sponsorship. Check out the event website for all the information. ccrew.accesscr.com.au/cpir-2025/

Do you take requests AAR? ...seems to be more molecular investigations to explain unknown phenotypical variation. Pls share your wisdom about these gene/GW linkages and their impact? www.jbc.org/article/S002...
doi.org/10.3389/fgen...

Cases make for thoughtfulness, i see attempting genuine efficacy in small pseudoexon pop as opposed ongoing barriers evident so far with each approved hotspot patch, seems to be advice of Matsuo in his history of exon skipping paper Feb 3. Can Sensei you review it ?

This disease has so many paradoxes on several levels, most ignored only independent expertise with skin in game co-designing for precision (and transparency of decisions however admirable, to discontinue)can change bad CT designs.

microsampling not invasive at all, way underused (am connected to norway led project to change in RD) unless volume for good assay exceeds collection?

try to figure that out before we go any further. And let’s be clear: there is precedence for *not* trying to exercise through illness: if someone had Duchenne’s muscular dystrophy and told you they couldn’t walk to the bathroom easily anymore, you wouldn’t ask them to do that 18/

Enjoying the celebration of 10-years of the John Walton Muscular Dystrophy Research Centre @newcastleuni.bsky.social Amazing to see how colleagues contributed to neuromuscular disease research, diagnostics, therapy and international networking!

My son has Duchenne. It resonated with me that his avatar first ran across country some distance every time he logged on.

It’s not unheard of to keep your diagnosis private in online communities. Vince (Captain Google)did it as member of the Ukulele Underground but not for so long. When he passed, it became known that he had Duchenne and the Underground produced this tribute. youtu.be/bZknGc7eDmI?...

Regenxbio $RGNX says gene therapy strengthened boys with Duchenne muscular dystrophy

Pivotal study started, top-line data could come early 2026. FDA accelerated approval filing to follow.

www.statnews.com/2024/11/18/d... $SRPT

Differentiating Juvenile Dermatomyositis from Muscular Dystrophy

A diagnostic approach to sooner diagnosis in juvenile dermatomyositis

In Arthritis & Rheumatology
acrjournals.onlinelibrary.wiley.com/doi/abs/10.1...
#Medsky