Views & Analysis Living with DMD: Inspiring each other’s futures Diagnosed with Duchenne muscular dystrophy (DMD) at three years old, Dr Jon Hastie grew up n
News Drug price controversies hit pharma's reputation Patient groups slam pharma over drug costs in latest survey.
News FDA clears Ionis drug as first therapy for rare disease FCS Ionis Pharma's Tryngolza (olezarsen) has become the first FDA-approved drug therapy for adults with rare disease familial chylomicronaemia syndrome.
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