iiinova — where your digital self keeps evolving

Annemieke Aartsma-Rus @oligogirl.bsky.social · 02/10/2026

#apaperaday The @worldmusclesociety.org #WMS2026 special continues with a pick from Stenler et al in @moltherapy.bsky.social nucleic acids on a comparison of antisense oligonucleotide (ASO) treatment in adult and very old mice. DOI 10.1016/j.omtn.2026.103017

110

Reposted by Annemieke Aartsma-Rus

DMM Journal @dmmjournal.bsky.social · 01/10/2026

Maaike van Putten @oligogirl.bsky.social & 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....

Characterization of dystrophin expression and histopathology.

112

Annemieke Aartsma-Rus @oligogirl.bsky.social · 30/09/2026

#apaperaday Today’s pick is @worldmusclesociety.org and givinostat themed to commemorate the Italfarmaco symposium I’ve spoken at today. The pick is by Phongavanh et al in @moltherapy.bsky.social nucleic acids combining exon skipping and givinostat in mdx52 mice. Doi 10.1016/j.omtn.2026.103047

101

Annemieke Aartsma-Rus @oligogirl.bsky.social · 18/09/2026

I am looking forward to attending & presenting at & learning from other presenters at #KSCardioGeneMed27 @keystonesymposia.bsky.social meeting in Banff next January. For more information: hubs.la/Q04xPBvR0 Abstract deadline: Sept 30 Fellowship deadline: Sept 30 Hope to see you there

053

Annemieke Aartsma-Rus @oligogirl.bsky.social · 18/09/2026

#apaperaday Last 1 before my holiday but back @worldmusclesociety.org conference. As announced due to teaching, the frequency was already reduced this week. Today's feature is a paper by Shahin et al on a late stage Duchenne patient at ICU in Case Reports in Critical Care. Doi 0.1155/crcc/9969987

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 15/09/2026

#apaperaday The paper looks smudgy because it was travelling with me the past days before I could read it. Yuzu looks dapper and is making confetti at her temporary home. Today's pick is by Mercuri et al, the plain language summary of the givinostat trial in DMD. doi 10.6084/m9.figshare.33582548

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 11/09/2026

Please note that next week due to teaching there will be intermittent #apaperadays depending on how much time I have. Then #apaperaday will be on a break until a @worldmusclesociety.org special starting September 30 and @n1collaborative.bsky.social and OTS specials starting on October 9. Stay tuned!

static.klipy.com

Coffee Break Time Animated Cup

ALT: Coffee Break Time Animated Cup

020

Annemieke Aartsma-Rus @oligogirl.bsky.social · 11/09/2026

#apaperaday Today's pick was already waiting a while back, but Yuzu discovered it pre-reading (picture evidence provided). I had to reprint 4 pages due to excessive confetti making 😇. It is by Feng et al in @pnas.org on ways to improve exon skipping in Duchenne models. doi: 10.1073/pnas.2606494123

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 10/09/2026

#apaperaday Today's pick is from the OTS affiliated journal Nucleic Acids Therapeutics by Mondragon et al on the evaluation of neuronal lysosome changes after exposure to antisense oligonucleotides (ASOs). doi: 10.1177/21593337261473518

140

Annemieke Aartsma-Rus @oligogirl.bsky.social · 09/09/2026

#apaperaday from home because of a public transport strike. Yuzu is happy with the selected pick and extra attention. The pick is from @worldmusclesociety.org journal Neuromuscular Disorders by Rajakumar et al on the analysis of 3 Duchenne mouse models. doi: 10.1016/j.nmd.2026.107396

111

Annemieke Aartsma-Rus @oligogirl.bsky.social · 08/09/2026

Documented counseling was there for only 26% of the patients with a psychosocial health concern. Of the rest 24% did have a referral but did not attend counseling (at least there was no recording of this). For 50% of patients with identified symptoms there was no referral.

000

Annemieke Aartsma-Rus @oligogirl.bsky.social · 08/09/2026

#apaperaday @worldduchenne.bsky.social #WDAD2026 themed again for the afterparty. A pick from Gupta et all on the use of psychosocial care among Duchenne patients in the USA - again related to the theme 'access'. doi: 10.1016/j.pediatrneurol.2026.06.022. Also Klaus is finally back home!

112

Annemieke Aartsma-Rus @oligogirl.bsky.social · 07/09/2026

#apaperaday #WDAD2026 @worldduchenne.bsky.social Wearing my WDAD t-shirt in Tuebingen to raise awareness. Today’s pick is related to access, the #WDAD2026 theme. It is from @frontiersin.bsky.social psychology by Alruthia et al in the impact of Duchenne in Saudi Arabia doi 10.3389/fpsyg.2026.1828571

121

Annemieke Aartsma-Rus @oligogirl.bsky.social · 04/09/2026

#apaperaday Today's pick is @worldduchenne.bsky.social #WDAD2026 themed & related to Duchenne & access. It is from @moltherapy.bsky.social nucleic acids by Geoffroy-Giraud et al on the development of antisense oligonucleotides (ASO) for rare variant exon skipping. DOI: 10.1016/j.omtn.2026.103048

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 03/09/2026

#apaperaday Today's pick is the first @worldduchenne.bsky.social #WDAD themed pick on the influence of community disadvantage of cognitive performance of Duchenne boys but also carrier mothers by Karra et al in Brain communications. doi: 10.1093/braincomms/fcag306

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 02/09/2026

#apaperaday Today's pick is on the platform technology approach and a stakeholder perspective on the change in EU medicine legislation for this topic by Oskam et al in Drug Discovery Today 10.1016/j.drudis.2026.104740 (and yes the origami turtle is serving as a platform for the micro-turtles).

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 01/09/2026

#apaperaday Today there will not be a 🧵because I have too many online meetings. However, there will be an @worldduchenne.bsky.social themed series starting later this week and early next week to commemorate the #WDAD2026 theme 'access' to look forward to!

010

Annemieke Aartsma-Rus @oligogirl.bsky.social · 31/08/2026

#apaperaday Today's pick is an invited letter to Clinical Translational Medicine by Guo et al on how a Duchenne rhesus monkey model can bridge the translational gap for therapy development. DOI: 10.1002/ctm2.70786

100

Annemieke Aartsma-Rus @oligogirl.bsky.social · 28/08/2026

#apaperaday Today's pick if from Human Gene Therapy by Siddiqui et al on using pretreatment of sirolimus for Elevidys (AAV micro-dystrophin gene therapy) treatment of Duchenne patients. doi: 10.1177/10430342261472609.

140

Annemieke Aartsma-Rus @oligogirl.bsky.social · 27/08/2026

#apaperaday Today's pick is a commentary from Kirschner and Bonneman on the publication by McDonald et al on the HOPE-3 demamiocel trial in Duchenne that featured in an earlier #apaperaday bsky.app/profile/olig... doi: 10.1016/S0140-6736(26)01656-9. lets see what the experts have to say :)

140

Annemieke Aartsma-Rus @oligogirl.bsky.social · 26/08/2026

#apaperaday Today's pick is by Gentil et al in @moltherapy.bsky.social nucleic acids on the effect of GDF5 on muscle stem cell (MUSC) proliferation and differentiation in the mdx mouse model. Doi: 10.1016/j.omtn.2026.103005

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 25/08/2026

#apaperaday Today's pick was selected by Yuzu as good confetti material. It is from @moltherapy.bsky.social Nucleic Acids by Vermeer et al, with a meta-analysis of adverse events of clinical trials using antisense oligonucleotides (ASOs). doi: 10.1016/j.omtn.2026.102976

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 24/08/2026

#apaperaday Today a new treasure hunt has initiated in the Department with micro-turtles. The pick is another hunt, by Fujimoto et al in @worldmusclesociety.org journal Neuromuscular disorders: the hunt for dystrophin isoform Dp40. Do it exist? DOI: 10.1016/j.nmd.2026.107390

100

Annemieke Aartsma-Rus @oligogirl.bsky.social · 20/08/2026

#apaperaday today's pick is from Hassan et al in Cureus with a case report of a Duchenne patient who developed fat embolism syndrome after elective surgery. Authors present the case to raise awareness of unexpected/unanticipated risks. DOI: 10.7759/cureus.112419

100

Reposted by Annemieke Aartsma-Rus

Oded Rechavi @odedrechavi.bsky.social · 19/08/2026

Lab meetings can be the worst, or the best! :) Here are 22 tips for improving them (including mine) www.nature.com/articles/d41... @nature.com

nature.com

Twenty-two easy ways to power up your lab meetings

Scientists share help and advice on how they got their research discussions flowing.

06624

Annemieke Aartsma-Rus @oligogirl.bsky.social · 19/08/2026

#apaperaday Today's pick is from @nejm.org by Samuelson-Jones - a letter about a patient with a cardiotoxic response after elevidys treatment. Authors show low levels of micro-dystrophin expression in muscle and heart. The letter is 2 pages, with 30 pages supplementary data doi: 10.1056/NEJMc2518477

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 17/08/2026

#apaperaday Today's pick is by Leffler et al on the impact clinical trial participation has on the quality of life of Duchenne patients and caregivers. Study executive by someone living with Duchenne. doi: 10.1007/s40290-026-00624-y.

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 14/08/2026

Our collaborative effort initiated by @erdera.bsky.social in collaboration with @n1collaborative.bsky.social on considerations for individualized ASO development for patients with neurodevelopmental diseases is now out in @cp-cellrepmed.bsky.social www.sciencedirect.com/science/arti...

sciencedirect.com

020

Annemieke Aartsma-Rus @oligogirl.bsky.social · 14/08/2026

#apaperaday Today's pick is by Vaknin et al. A preprint from @moltherapy.bsky.social nucleic acids on Safesense, a curated, open access database on adverse events (or lack thereoff) of antisense oligonucleotides (ASOs) tested in humans. DOI: 10.1016/j.omtn.2026.103035

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 13/08/2026

#apaperaday Today's pick is a 23 year old mini-review from Jim Ervasti, one of the individuals involved in the identification of proteins in the dystrophin-glycoprotein complex. The review is on the costameres - interesting to read with current day's knowledge. doi: 10.1074/jbc.R200021200

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 12/08/2026

#apaperaday Back after a day off with a pick from Ikelaar et al in @journalnd.bsky.social on the effects of intermittent steroid use on scoliosis, bone density and vertebral fractures in Duchenne patients. For transparency: work from LUMC, but I was not involved. doi: 10.1177/22143602261472975

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 10/08/2026

#apaperaday Today's pick is from Clinical Pharmacology Drug Development by Mercuri et al on a study in healthy volunteers with therapeutic and supratherapeutic doses of givinostat to assess the impact on QT intervals. doi: 10.1002/cpdd.70047.

210

Annemieke Aartsma-Rus @oligogirl.bsky.social · 07/08/2026

#apaperaday Today's pick is from Muscle and Nerve by PIetrusz et al on the pulmonary function of a large UK cohort of adult Duchenne patients who were continued or stopped steroids in adulthood vs naive patients. doi: 10.1002/mus.70312

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 06/08/2026

#apaperaday Today's pick is a case report from @eshg.bsky.social journal @ejhg-journal.bsky.social by Willemse et al on an ALS patient with a variant of unknown significance in SOD1 who was treated with tofersen. doi: 10.1038/s41431-026-02208-5

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 05/08/2026

#apaperaday Today's pick is the paper from Yang et al in @nature.com about a base editing approach that was tested in mice and monkeys and it later turned out also in a human (with lethal consequences). doi: 10.1038/s41586-026-10113-6

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 04/08/2026

#apaperaday Today's pick is from Journal of chemical information and modelling by Hwang et al on a framework to predict gapmer ASO activity. Yuzu was more interested in her ball collection than confetti making today. doi: 10.1021/acs.jcim.6c01314

100

Annemieke Aartsma-Rus @oligogirl.bsky.social · 03/08/2026

#apaperaday Today's pick is from @natmed.nature.com by Kim-McManus et al on the treatment of 2 patients with developmental and epileptic encephalopathy with an allele-selective individualized antisense oligonucleotide (ASO) targeting SCN2A DOI: 10.1038/s41591-026-04527-y

100

Annemieke Aartsma-Rus @oligogirl.bsky.social · 31/07/2026

#apaperaday Today's pick is by McDonald et al from @thelancet.com the publication on the HOPE-3 demamiocel trial. Publication was well timed with the FDA meeting, but did not positively impact the votes. doi: 10.1016/S0140-6736(26)01385-1

130

Annemieke Aartsma-Rus @oligogirl.bsky.social · 27/07/2026

#apaperaday we are back from the NMSS2026 in India, with a pick today from @worldmusclesociety.org journal Neuromuscular Disorders by Conrad et al on a 9 patient case series of female dystrophinopathy with severe cardiomyopathy. doi: 10.1016/j.nmd.2026.106484.

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 24/07/2026

#apaperaday Final #NMSS2026 Indian Summerschool special is a pick by Vorwald et al in the journal of physiology on the effect of a high fat high sugar diet on mdx mice with or without steroid treatment. Doi 10.1113/JP289832

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 23/07/2026

#apaperaday India Summerschool special part 4 is by Engebrecht et al in muscle and nerve on the development and validation of a caregiver reported outcome measure for Duchenne muscular dystrophy patients (DMDCR-HI). Doi 10.1002/mus.70327.

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 22/07/2026

#apaperaday india summer school special part 3 is a pick from cardiology therapy by Veerapandiyan et al on the long term cardiac outcomes of young Duchenne patients treated with elevidys (the FDA approved AAV74 micro-dystrophin). doi: 10.1007/s40119-026-00457-5

220

Annemieke Aartsma-Rus @oligogirl.bsky.social · 21/07/2026

#apapreaday #NMSS2026 summerschool special part 2 is by Østergaard et al in @narjournal.bsky.social on the development of bicycle peptides targeting transferrin receptor 1 (TfR1) to deliver oligonucleotide therapies to muscle. Doi 10.1093/nar/gkaf270.

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 20/07/2026

#apaperaday India neuromuscular summer school #NMSS specials coming your way this week from Bangalore with a first pick from journal of cachexia sarcopenia and muscle by Degan et al on blood biomarkers that correlate with function in Duchenne patients DOI: 10.1002/jcsm.70320

100

Annemieke Aartsma-Rus @oligogirl.bsky.social · 17/07/2026

#apaperaday Today's pick is by Li et al in @narjournal.bsky.social on a bottlebrush polymer (PEG) to deliver an antisense oligonucleotide (ASO) to skeletal muscle in a myotonic dystrophy mouse model. Its called bottlebrush due to the shape (Yuzu does an impression). doi: 10.1093/nar/gkag619

120

Annemieke Aartsma-Rus @oligogirl.bsky.social · 16/07/2026

#apaperaday Today's pick is a cautionary tale case study presented by Jiang et al in Translational Pediatrics, about a patient with female dystrophinopathy who presented with elevated 'liver' (but really muscle damage) enzymes. doi: 10.21037/tp-2026-1-0144.

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 15/07/2026

#apaperaday Today's pick is a review from @ijmsc.bsky.social by Sorokina et al on fibro-adipogenic progenitor (FAP) cells. A cell type that was long overlooked but which has important functions for skeletal muscle health. DOI: 10.3390/ijms27115016

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 14/07/2026

#apaperaday Today's pick is from Nucleic Acid Therapeutics by Evequoz et al on the abcDNA backbone chemistry, testing it for a gapmer RNase H approach after showing earlier that it can do exon skipping. I was involved in the exon skipping work but not in this effort. doi: 10.1177/21593337261417504

110

Annemieke Aartsma-Rus @oligogirl.bsky.social · 13/07/2026

#apaperaday Today's pick is from @narjournal.bsky.social genomics and bioinformatics by Vlasenok et al on the use of alternative intronic polyadenylation to escape nonsense mediated decay. Yes those pigs indeed mean to represent a poly-A-pig-tail. doi: 10.1093/nargab/lqag052

120