Ultragenyx's strong revenue growth, diverse streams, and late-stage pipeline make it a standout in rare disease and gene ...

I care deeply because this chronic disease is mine. It lives inside me. It’s radically changed my life and the life of my ...

Parents of children with spinal muscular atrophy (SMA), a rare disease, welcomed the announcement by the Indian pharma ...

Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~~ ...

The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a ...

Ipsen and its fellow French partner Genfit are competing with Intercept and Advanz’s Ocaliva, which was approved in 2016 and ...

IgG4-related disease (IgG4-RD) is a systemic fibroinflammatory condition marked by the accumulation of IgG4-positive plasma ...

Crofelemer, a novel plant-based anti-secretory prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines ...

The biotech is developing an experimental drug for the most common of a family of disorders all caused by errors in ...

Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than ...