Poly(GP) proteins: a potential pharmacodynamic marker in ALS and FTD

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The genetic factor chromosome 9 open reading frame 72, C9ORF72, has been closely associated not only with the development of ALS, but also with frontotemporal dementia (FTD) characterized by a progressive cognitive decline, altered personality and impairment in language (1,2).