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 ...

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 ...