NEURO360-aligned research from Washington University School of Medicine reports encouraging long-term results for tofersen, an FDA-approved therapy for a rare genetic form of ALS caused by SOD1 mutations. Extended treatment slowed disease progression and improved survival, with about one-quarter of participants experiencing stabilization or gains in strength and mobility.

These findings highlight the potential of targeted, gene-based therapies to change the trajectory of ALS and inform future treatments for other neurodegenerative diseases.

Read the full story on the WashU Newsroom website by clicking here.