Given the importance of PGRN in ALS development, AL001, a human monoclonal antibody that blocks the sortilin-PGRN interaction to prevent the degradation of PGRN, is currently being investigated in a Phase II study to evaluate its safety, pharmacokinetics, and pharmacodynamics in ALS-C9orf72.547 As other neurodegenerative disease, drugs targeted to some key molecules involved in inflammatory signaling pathways, including TLR2, IL-1R, IL-6R, JAK and receptor-interacting serine/threonine-protein kinase 1 (RIPK1), have also been developed. This evidence concerns the gene SORT1 and amyotrophic lateral sclerosis.