The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to DTx Pharma’s DTx-1252, an investigational small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMT1A is a progressive, neuromuscular, autosomal-dominant disease that leads to life-long loss of muscle function and disability. “We are pleased to receive Orphan […]
