Acoramidis, an oral therapy, is now approved to reduce cardiovascular-related deaths and hospitalizations in adults with hereditary or wild-type ATTR-CM.
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.
Crinecerfont, a first-in-class therapy, improved androgen control and reduced reliance on high-dose glucocorticoids for patients with congenital adrenal hyperplasia.
The FDA has approved a novel therapy for patients with hemophilia A or B without inhibitors. Clinical trials showed it significantly reduces bleeding episodes by targeting a new protein in the blood...
Two randomized, double-blind studies demonstrated that a new antipsychotic treatment for adult patients with schizophrenia may lead to a meaningful reduction in symptoms.
In June, following a placebo-controlled trial, the FDA approved pembrolizumab with chemotherapy to treat patients with primary advanced or recurrent endometrial carcinoma.