Novel insights and therapeutic candidates continue to emerge in neurological disease research. Biogen advanced its next-generation spinal muscular atrophy antisense oligonucleotide salanersen into registrational studies following positive phase 1 data, offering hope for patients inadequately treated by Zolgensma. City of Hope launched HopeLLM, a generative AI platform to optimize oncology care and clinical trial matching. CAR T and RNAi therapies targeting glioblastoma and TDP-43 proteinopathies respectively showcase progress in tackling neurodegnerative and brain cancers. These developments highlight expanding strategies combining gene therapy, AI, and molecular targeting to improve neurological outcomes.