GA-VAX-01 shows promising results for C9orf72-associated ALS and FTD
July 10, 2024
Many patients with amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD) harbor the (G4C2)n pathogenic repeat expansion in the C9orf72 gene, which leads to aggregating dipeptide proteins, mainly poly-glycine-alanine (poly-GA).