Latest News

Second Patient Treated with HG-CT-1 CAR-T Therapy Successfully Passes Initial Safety Evaluation LONDON, UK – Hemogenyx Pharmaceuticals plc is pleased to announce that the second patient has been successfully treated in the ongoing Phase I clinical trial of HG-CT-1, the Company’s proprietary Chimeric Antigen Receptor T-cell (CAR-T) therapy for relapsed/refractory...
Among patients with hemophilia A who receive primary prophylaxis, quality of life (QoL) may be lower compared with the general population, according to research published in Haemophilia. Among patients with severe hemophilia, prophylaxis with clotting factor replacement therapy is the standard treatment, though patients vary in when they initiate therapy. In...
Adult men with hemophilia B had annual health care costs that totaled more than 25-fold higher than matched controls, demonstrating the burden of this illness in research published in Blood Advances. The study authors used a claims-based algorithm to identify 454 male patients with hemophilia B and compared them to 454...
CHARLOTTESVILLE, Va. – HemoShear Therapeutics, Inc., a clinical stage company developing treatments for rare metabolic disorders, today announced that the first two patients have been dosed in the HERO (HElp Reduce Organic Acids) Phase 2 clinical trial of HST5040, an oral small molecule drug being investigated for the treatment of patients with methylmalonic...
FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that Hepatology published an analysis demonstrating a statistically significant improvement in six-year event-free and transplant-free survival in patients with Alagille syndrome (ALGS) treated with LIVMARLI® (maralixibat) oral solution when compared with a natural history control group (p<0.0001). The analysis...
Changchun, China – A new study published in the journal eGastroenterology provides an updated assessment of the diagnosis, pathophysiology, and treatment of hepatorenal syndrome-acute kidney injury (HRS-AKI). This severe and often fatal condition can occur in patients with cirrhosis. HRS-AKI is a functional and progressive kidney failure that is potentially...