FSHD is a genetic autosomal dominant muscular dystrophy that results in significant disability and is associated with an immune component. The early onset FSHD phenotype is characterized by more severe, rapidly progressive muscle involvement. ATYR1940 is a Physiocrine-based protein that is nearly identical to human histidyl-tRNA synthetase and has been shown in preclinical studies to modulate immune responses in skeletal muscle. ATYR1940-C-003 is a multicenter, open-label study that evaluated the safety, tolerability, and pharmacodynamic activity of intra-patient dose escalations of intravenous ATYR1940 administered once weekly (qw).

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