Genetic and Congenital

Long-term study on givosiran shows promising results for acute intermittent porphyria

Givosiran, an RNA interference therapy, has a strong long-term safety profile and significant clinical efficacy for treating adults with acute intermittent porphyria, according to results of a study. Over a follow-up period of up to 48 months, patients experienced dramatic reductions in the frequency of porphyria attacks and hemin usage,...