Genetic Disorders

Plozasiran, a first-in-class RNA interference therapeutic, reduced triglycerides by 80% and lowered the risk of acute pancreatitis by 83% in patients with familial chylomicronemia syndrome (FCS), earning...

The FDA has approved concizumab-mtci, a daily subcutaneous treatment to reduce bleeding for patients with hemophilia who have clotting factor inhibitors.

The FDA approved givinostat, a nonsteroidal oral medication, for treating patients 6 years of age or older with Duchenne muscular dystrophy.

The FDA approved two gene therapies, the first of their kind, to treat patients aged 12 years and older with sickle cell disease.

The FDA has approved a treatment for Friedreich ataxia after the results of randomized, placebo-controlled study indicate a strong effectiveness in treatment.