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Acta Neuropathologica Communications|May 24, 2025
Viral-mediated knockdown of Atxn2 attenuates TDP-43 pathology and muscle dysfunction in the PFN1<sup>C71G</sup> ALS mouse modelZachary C E Hawley, Xueying Li, Dora Bodnar, et al.Cell|June 23, 2026
An emergent disease-associated motor neuron state precedes cell death in ALSOlivia Gautier, Jacob A Blum, Thao P Nguyen, et al.Journal of Medicinal Chemistry|March 17, 2026
Discovery and Optimization of Thienopyrazine RNA-Splicing Modulators for the Treatment of Huntington's DiseaseChaofan Xu, Neeta Abraham, Nupur Bansal, et al.Molecular Therapy. Methods & Clinical Development|December 17, 2025
Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following AAV gene therapyBranka Grubor, Kate L Henry, Su Jing Chan, et al.Pageof 2