유전자 침묵제가 ATTR 심근병증 환자에게 인상적인 결과를 보여
유전자 침묵제 vutrisiran은 ATTR 심근병증 환자에서 사망률과 심혈관 사건 재발을 크게 감소시켰다.
GSK's Nucala Asthma Drug Meets Primary Endpoint in Late-Stage Study for Treating Chronic Obstructive Pulmonary Disease (COPD) in Smokers
Nucala, GSK's asthma drug, met the primary endpoint in a late-stage study, demonstrating a lower annualized rate of moderate or severe exacerbations in COPD patients compared to placebo.
Approval of Givinostat, the First Non-Steroidal Therapy for Duchenne Muscular Dystrophy, Awaits Decision in Europe
The FDA has approved givinostat, the first non-steroidal therapy for Duchenne muscular dystrophy, and the drug is now under review for marketing authorization in Europe, where it could represent a significant milestone in the treatment of this debilitating condition.
Approval of Danicopan, a Novel Complement Inhibitor, for Paroxysmal Nocturnal Hemoglobinuria in Europe
The European Commission has approved danicopan, a first-in-class complement factor D inhibitor, as an add-on therapy for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and continuing clinically significant extravascular hemolysis despite treatment with standard complement blockade.
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