Authors discuss that future work should include human IPSC lines as the mouse model is rather artificial and also has a very quick onset and loss of motor neurons so seeing treatment effects might be challenging
Third #OTS2026 special coming on Monday

#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

Authors also describe in vitro studies that have already been done to assess ASO safety. The Hagedorn work showing that Calcium oscillations in cultured cells correlated with in vivo toxicity in mice after local CNS delivery of ASOs is mentioned first (there will be a talk from Hagedorn at #OTS2026)

#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.