AnnemiekeAartsma-Rus
@oligogirl
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Translating science from bench to bedside and from jargon to lay language
Haarlemmermeer, Nederland
Joined March 2016
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Read a very interesting paper today in @LiebertPub Human Gene Therapy by Laforet at al on TMA after AAV gene therapy. DOI: 10.1089/hum.2024.156 Full story on Bluesky 👇
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Today on @bluesky I discuss the publication on Brogidirsen - the first in human clinical trial is now published in @CellReports
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Looking forward to seeing everyone there: old OTS friends and new OTS friends
The regional delivery grant event First Nucleic Acid Therapeutics Regional Meeting is approaching SOON! Join us for the meeting on February 18-21, 2025 in Bengaluru, India at the Jawaharlal Nehru Centre for Advanced Scientific Research (JNCASR). #ots
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Several of my followers suggested this paper from @NatureComms by Millozi et al on aptamer covered nanoparticles to deliver to mouse muscle stem cells for #apaperaday Today it happened! 👇
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RT @TREAT_NMD: Register Your Interest: SMA/DMD Educational Masterclasses in Prague! Join TREAT-NMD® for expert-led masterclasses tailored…
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This will be a unique opportunity for those working on Duchenne in industry to get an induction in all things Duchenne (genetics, pathology, tools and models, care and management, trial design and outcome measures etc etc). Highly recommended!
TREAT-NMD is thrilled to announce an in-person, expert-led one-day symposium on Duchenne Muscular Dystrophy (DMD) in Prague, Czech Republic, on July 11th, 2025! This event is perfect for industry professionals new to the field of Duchenne and those seeking a comprehensive overview of this complex area. 💡 Topics Covered ✅ Clinical aspects of the disease ✅ Disease management ✅ Genetic causes and pathophysiology ✅ Preclinical tools and models ✅ DMD therapeutic landscape ✅ Clinical trial design ✅ Outcome measures for therapy development 🎯 Objectives 🔹 Equip participants with practical insights to apply in their work 🔹 Create a collaborative platform for sharing ideas, addressing challenges, and celebrating successes 📢 Limited Places Available! Be the first to know when registration opens—complete the expression of interest form now! 👇 #Duchenne #TREATNMD #NeuromuscularDiseases #DMD #LifeSciences #Prague2025
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1st paper of the year is out! Technically it is a paper from last year of course: a review on histone deacetylase inhibitors (specifically givinostat) and how they can impact on pathology of Duchenne. Disclaimer: open access is paid for by @italfarmacoes
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RT @OTSociety: Save the date! Join us at the 21st Annual Meeting of the Oligonucleotide Therapeutics Society, October 19-22, 2025, in Budap…
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Interested in reading what I have to say about the caregiver impression study that was done for the Elevidys EMBARK gene therapy study? You can read it on @bluesky in #apaperaday
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RT @TREAT_NMD: Duchenne Muscular Dystrophy Therapeutic Pipeline Update We have recently completed our annual update to the Duchenne Muscul…
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Interested in the #apaperaday on the qualification of the first digital endpoint (stride velocity 95th percentile, SV95C) by Servais et al please see
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#apaperaday Today's pick is a short communication from @pediatric Neurology by Duvuru et al about 2 boys with Duchenne who developed acute liver injury after treatment with Elevidys. If you want to read the complete thread, go to
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RT @TREAT_NMD: 📣 Registration of Interest Open: TREAT-NMD Expert Masterclasses for SMA and DMD Please register* to attend our Expert mas…
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Note that #apaperaday has moved to @bluesky Get your daily dose of papers there from Today we feature the Becker rat model developed by the group of Fred Relaix, which develops cardiomyopathy
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Are you missing your daily dose of #apaperaday? You can still get it on @bluesky where I post under @oligogirl.bsky.social Today a paper on reference genes for mouse muscle tissues on various Duchenne models.
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Authors end by saying that rare diseases can be actionable, e.g. the N=1 treatment development for genetic diseases (@n_lorem @N1Collaborative ). I of course agree with this. I also agree there will be more than 10,000 rare diseases and that diagnosing the very rare is tough
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