Fulcrum had identified inhibitors of p38/ mitogen activated protein kinase (MAPK) as powerful inhibitors of DUX4 expression. DUX4 is the gene that is the root cause of FSHD, a progressive muscle ...

EPI-321 is a one-time gene-modulating therapy designed to silence aberrant expression of DUX4, a gene that is incorrectly activated in FSHD and leads to progressive muscle degeneration.