In contrast, untreated symptomatic FTD-GRN patients typically experience increasing NfL levels, underscoring the potential of PBFT02 in slowing disease progression. As of December 2024, Passage Bio is progressing to evaluate a 50 percent lower Dose 2 in additional cohorts of FTD-GRN and FTD-C9orf7...
Loss-of-function mutations in the progranulin gene (GRN), which encodes progranulin (PGRN), are a major cause of frontotemporal dementia (FTD). GRN-associated FTD is characterized by TDP-43 inclusions and neuroinflammation, but how PGRN loss causes disease remains elusive. We show that Grn ...