In this condition, a mutation causes a gene called DUX4 to switch on. The protein it makes is "poison" to the muscles, says ...

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that ...

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 an investigational 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 ...

FSHD is a devastating muscle-wasting disease caused by the activation of an abnormal gene which increases the toxic build-up of the DUX4 protein in muscle cells, resulting in loss of strength in ...