End the Legacy NEALS 2025 Report
In three years of data, Tofersen led to 25% improvement on the ALS Functional Rating Scale, and early treatment in fast progressors added about 1.5 years of survival. A pause on an adeno-associated virus (AAV) gene therapy for SOD1 ALS shows safety risks in new approaches. New early-stage trials, like ALS My Match and the UK Experts platform, test small biomarker-guided groups; the first study uses Digoxin in a C9orf72 ALS cohort.
Research#Amyotrophic Lateral Sclerosis (ALS)#Motor Neurone Disease (MND)#C9orf72#Tofersen#SOD1