#apaperaday From Denver where today we have N of 1 collaborative ancillary meetings today and tomorrow we have our annual meeting prior to #OTS2026
Today’s pick is by Heinsinger et al in Neurology of Disease on splicing modulation of ataxin 2 as a strategy to treat ALS
Doi 10.1016/j.nbd.2026.107582

#apaperaday Today's pick is on travel day to N1C and OTS meeting with flight rebook stress. But courtesy of Japan jetlag, I was awake on time to read it. The pick is from @moltherapy.bsky.social nucleic acids by Colar Zanjko et al on in vitro toxicity models for CNS ASOs. 10.1016/j.omtn.2026.103062.

#apaperaday @worldmusclesociety.org #WMS2026 belated special part 4 is by Benning et al on a meta analysis on survival of Duchenne patients published in Journal of neurology DOI 10.1007/s00415-026-14121-4.
Note that tomorrow we start an #OTS2026 oligonucleotide therapeutics society meeting special.

#apaperaday @worldmusclesociety.org #WMS2026 (belated) special part 3 is an extensive one: the ENMC report on safety aspects for AAV gene therapy in neuromuscular disease patients by Orbach et al published in @worldmusclesociety.org journal neuromuscular disorders Doi 10.1016/j.nmd.2026.107371.

#apaperaday I am aware there have not been daily paper 🧵during #WMS2026. The problem was that the wifi was as bad as the meeting was good, which interfered with tweeting. Yesterday was catchup day after returning from WMS (emails also suffered from the poor wifi) but today #apaperaday has a restart

Authors could show that in adult treated mice there was a reversal of transcript imbalance, but not for the aged mice after pPMO treatment (they discuss later that this has not been observed before by others – including Wednesday's #apaperaday where givinostat was required to achieve this)

#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

#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

#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

#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

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!

#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

#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

#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

#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!

#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

#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