Multiple studies highlight advances in understanding and treating neurodegenerative diseases. A new activator DNL343 targets eIF2B to address ALS and TDP-43 proteinopathies. Novel gene therapies progress for pediatric acute myeloid leukemia and bestrophin-1 inherited retinal disease. Research links iron mismanagement in Parkinson’s disease to disease mechanisms. Additionally, AI-driven blink analysis offers a non-invasive method to track Parkinson’s clinical fluctuations, enhancing disease monitoring and management strategies.