Translating science from bench to bedside and from jargon to lay language
Haarlemmermeer, Nederland
Joined March 2016
- Tweets22.3K
- Following450
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AnnemiekeAartsma-Rus retweeted
⏳ The deadline is approaching!
Want to expand your knowledge, connect with experts and help drive progress in rare diseases?
Apply for the Open Academy x ERDERA Schools 2027 before 16 October.
📍 Barcelona, 7-10 June 2027
🔗 Apply now: loom.ly/uZ0Iz8M
#ERDERA
AnnemiekeAartsma-Rus retweeted
Maaike van Putten @oligogirl & co develop four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.
Read the Resources & Methods article for free: doi.org/10.1242/dmm.052875
ALT Characterization of dystrophin expression and histopathology.
AnnemiekeAartsma-Rus retweeted
This article is a @ReviewCommons transfer. Find out more about Review Commons and the transfer to affiliate journals including @DMM_journal.
reviewcommons.org/
Hope you all had a good @DuchenneDay #WDAD2026. I was in Germany for a meeting, but had Duchenne access themed 🧵on Bluesky. Enjoy
bsky.app/profile/oligogirl.b…
bsky.app/profile/oligogirl.b…
AnnemiekeAartsma-Rus retweeted
On #WorldDuchenneAwarenessDay, we’re highlighting this year’s theme: Access Changes Lives. We’re committed to advancing education, knowledge and best practice to support better care for people living with Duchenne.
#WDAD2026 #Duchenne #DMD
Are you excited for @DuchenneDay #WDAD2026? Themed #apaperaday picks have started today on bluesky - more will follow until September 8.
bsky.app/profile/oligogirl.b…
Our collaborative effort initiated by @ERDERA_org in collaboration with @N1Collaborative on considerations for individualized ASO development for patients with neurodevelopmental diseases is now out in @CellRepMed
sciencedirect.com/science/ar…
Thank you very much for the hospitality @StJohnsResearch and I feel priviliged to have been part of #NMSS2026
Day 3 featured insightful discussions on gene therapy, novel genomic insights, drug repurposing, and translational research, highlighting how cutting-edge science is transforming the future of care for neuromuscular and rare genetic disorders.
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#NMSS2026 #Day3 #TranslationalResearch #GeneTherapy #RareDiseases #NeuromuscularDiseases #MedicalResearch #HealthcareInnovation #StJohnsMedicalCollege #ResearchForHope
#NMSS2026 @nmss2026 #apaperaday special part 3 out now. About cardiac follow up in elevidys treated patients to commemorate the translational symposium we have today.
bsky.app/profile/oligogirl.b…
#PPMD2026 #apaperaday specials on @bluesky 👇Another one coming tomorrow. If you are at the meeting: see you soon.
bsky.app/profile/oligogirl.b…
bsky.app/profile/oligogirl.b…
Tomorrow I will travel to #PPMD2026 @ParentProjectMD My Duchenne themed #apaperaday threads can be followed on @bluesky
bsky.app/profile/oligogirl.b…
AnnemiekeAartsma-Rus retweeted
Are you involved in neuromuscular care?
Applications are now open for the Adult Neuromuscular Disease Masterclass 2026 (European-based HCPs)
This 1,5 day masterclass is expert-led, and includes patient-centred learning.
Link to more info and application below
AnnemiekeAartsma-Rus retweeted
To mark our 20th anniversary, we announce The Past and Future of Translational Research in Neuromuscular Disease May 2027.
Building on our 2026 conference, this event reflects 20 years of progress and the future we will shape together. Register your interest to attend below.
AnnemiekeAartsma-Rus retweeted
🧬New Perspective from @oligogirl & co!
📄Guidance on communication and informed consent with patients and their families for experimental individualized treatments
cell.com/ajhg/fulltext/S0002…
And also the link for the @ojrarediseases publication on funding models for individualized therapy development link.springer.com/article/10…
There have been 2 @N1Collaborative papers out recently. One on funding models in @ojrarediseases and one in @AJHGNews
Guidance on communication and informed consent with patients and their families for experimental individualized treatments - ScienceDirect
There have been 2 @N1Collaborative papers out recently. One on funding models in @ojrarediseases and one in @AJHGNews
Guidance on communication and informed consent with patients and their families for experimental individualized treatments - ScienceDirect
New paper out! It was an @biorxivpreprint for a while already, but now also formally published in @JPathology Nice collaboration within LUMC, but also with experts from USA and UK and featured in @LauraHeezen thesis (Dr Heezen now)
pathsocjournals.onlinelibrar…
New paper in @journal_nd Lessons learned from 15 years @TREAT_NMD Advisory Committee for Therapeutics (TACT).
As people who have written reports with large numbers of authors will recognize, it was a long time coming, but it is finally out now🥳
journals.sagepub.com/doi/pdf…
AnnemiekeAartsma-Rus retweeted
From the JND special issue on RNA therapeutics:
Aartsma-Rus & Takeda examine the development of ASO therapies for #Duchenne muscular dystrophy and #SMA.
Read the paper here: buff.ly/rJJb5N7
#Neuromuscular #RNAtherapeutics #DMD #SMA
