After 27 years in business, Cytokinetics hopes to pit its own cardiac myosin inhibitor against one it initially developed—now owned by Bristol Myers Squibb—in a market worth billions. Aficamten has a ...

Insmed’s oral drug Brinsupri did not ease the burden of sinus symptoms in a mid-stage study of chronic rhinosinusitis without nasal polyps, forcing the New Jersey biotech to discontinue its program in ...

A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 ...

BioMarin Pharmaceutical has faced a rocky road, promising and then backing off revenue targets and cutting assets that have underperformed. But Amicus’ rare disease portfolio is already bringing in ...