OrphanPulse

A week defined by first-in-human starts across rare genetic disease, led by antisense entering Dravet syndrome from the field that already rewrote spinal muscular atrophy. Around it, first patients dosed in rare vascular and cardiomyopathy programs with near-empty competitive fields, a first-in-class readout in heparin-induced thrombocytopenia, and orphan designations that quietly reshape the economics behind them.