Science on Target: Q3 Publications from our Innovation Ecosystem

Antisense Oligonucleotide Tofersen Distribution in the Central Nervous System of SOD1-ALS Autopsy Tissue Donors. Guise AJ, Sellon MT, Roemer SF, Monine M, Comfort Harris NT, […] Plowey ED.  JAMA Neurol. 2026 Jul 6:e262195. doi: 10.1001/jamaneurol.2026.2195. Epub ahead of print. PMID: 42406382; PMCID: PMC13338845.

Using human 3D organoid models to gain mechanistic insight in motor neuron diseases. Frizzi B, Barbosa Correia AM, Faravelli I, Van Den Bosch L, Corti S. Nat Rev Neurosci. 2026 Jul 6. doi: 10.1038/s41583-026-01057-x. Epub ahead of print. PMID: 42410005.

When network biology meets human genetics. Zhang S.  Cell Genom. 2026 Jul 8;6(7):101299. doi: 10.1016/j.xgen.2026.101299. PMID: 42419258; PMCID: PMC13347932.

Longitudinal plasma proteomics predict phenoconversion to clinically manifest ALS. Ran X, Wuu J, Qin ZS, McDermott MP, Cooper-Knock J, Li Y, […] Benatar M. . Nat Med. 2026 Jul 27. doi: 10.1038/s41591-026-04528-x. Epub ahead of print. PMID: 42509371.

Intron retention in health and amyotrophic lateral sclerosis. Wang CY, Taylor S, Pandya VA, Clarke BE, Pal K, […] Patani R. Brain. 2026 Aug 3;149(8):2604–18. doi: 10.1093/brain/awag142. Epub ahead of print. PMID: 42543164; PMCID: PMC13431790.

Quantitative Analysis of Axonal Degeneration and TDP-43 Aggregation in Compartmentalized Human iPSC-Derived Motor Neuron–Myotube Co-cultures. Subramaniam, A. G., de Andrade Gensas, L. K., Gradus-Pery, T. and Perlson, E. (2026). Bio-protocol 16(17): e5801. DOI: 10.21769/BioProtoc.5801.

Functional Activity of TDP-43: A Direct Biomarker for ALS. Sonkar, K.S.; Levi D’Ancona, V.; Cramp, J.; Shilling, H.; Giles, E.; […] Rothstein, J.D.; et al.  Biosensors 2026, 16, 446. https://doi.org/10.3390/bios16080446

The EQ-5D-5L in amyotrophic lateral sclerosis: an international longitudinal exploration as part of ALS-CarE. McFarlane, R., Anneser, J., Al-Chalabi, A., Chiò, A., Mcdermott, […] Hardiman, O. (2026). Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 1–10. https://doi.org/10.1080/21678421.2026.2715921

Evidence for Reactivation of Embryonic Cytoskeletal Remodeling Programs, silenced decades earlier, in Amyotrophic Lateral Sclerosis. Steven Lehrer.  14 August 2026, PREPRINT (Version 1) available at Research Square [https://doi.org/10.21203/rs.3.rs-10690389/v1]

Progress towards a biotypic biomarker profile for amyotrophic lateral sclerosis-frontotemporal spectrum disorders. Strong MJ, Donison N, Al-Chalabi A, Boeve B, Finger EC, […] Wolk DA.  Brain. 2026 Sep 5:awag297. doi: 10.1093/brain/awag297. Epub ahead of print. PMID: 42698276.

TDP-43 dysfunction induces cryptic circular RNAs in ALS/FTD. Dario Dattilo, Flaminia Pellegrini, Simone Barattucci, […] Sanjay Chandriani, Michael Ward, Pietro Fratta. bioRxiv 2026.09.17.752342; doi: https://doi.org/10.64898/2026.09.17.752342

Genetic ALS-Progressive Sleep Defects Precede the Onset of Clinical Disease. Lang C, Guillot SJ, Knehr A, Weydt P, Dorst J, Tumani H, Ludolph AC, Dupuis L, Bolborea M. Eur J Neurol. 2026 Sep;33(9):e70755. doi: 10.1111/ene.70755. PMID: 42765595; PMCID: PMC13591987.

Minding the gates: dual cell-autonomous and non-cell-autonomous mechanisms of TBK1 in ALS/FTD. Smeyers J and Mordes DA (2026)  Front. Neurosci. 20:1941293. doi: 10.3389/fnins.2026.1941293

LRSAM1, an E3 ubiquitin ligase, modulates TDP-43 proteostasis in amyotrophic lateral sclerosis. Shirakawa T, Mikuriya S, Inoue Y, Murasaki K, Salinas F, Takegawa-Araki T, Tamura M. Brain. 2026 Sep 28:awag334. doi: 10.1093/brain/awag334. Epub ahead of print. PMID: 42803299.

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