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Poly(GP) proteins as a potential pharmacodynamics marker in ALS and FTD. Gendron and colleagues showed that the testing of poly(GP) proteins in the CSF could be used to validate the efficacy of clinical trials for the treatment of c9ALS or c9ALS-FTD. ALS, amyotrophic lateral sclerosis; CSF, cerebrospinal fluid; RBPs, RNA-binding proteins; RAN, repeat-associated non-ATG; c9ALS, C9ORF72-associated ALS; FTD, frontotemporal dementia.
