LONDON, UK – Hemogenyx Pharmaceuticals plc (LSE:HEMO) is pleased to announce that it has been informed by the U.S. Federal Food and Drug Administration (FDA) that it has lifted the clinical hold on the Investigational New Drug (IND) application for HEMO-CAR-T for the treatment of acute myeloid leukemia (AML). The FDA...
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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...
Sugar Land, Texas – Over the past two decades, there has been a significant increase in the incidence of primary liver cancer in the USA, with higher rates observed in men. Its burden increases with age and disproportionately affects men, with mortality rates three times higher in men than in...
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...
MILFORD — Amanda Pitre, 30, lives with her parents and can only work part-time because she has an autoimmune disease that creates constant, burning pain as if a “blowtorch” was lit inside her. But Pitre, 30, who was born weighing only 2 pounds, 2 ounces, has begun forging a path to...
Wilson’s disease is a rare disorder of copper metabolism, an “orphan disease” – a condition so uncommon that its diagnosis and treatment had been neglected. The disease was recognised by the British neurologist Kinnear Wilson in 1912, but a simple means of detecting it did not exist until the 1950s,...
New York, NY — The Hereditary Neuropathy Foundation (HNF) announced today the formation of a Research Consortium (HNF-RC) designed to speed the development of new therapies for Charcot-Marie-Tooth disease (CMT), which affects approximately 1 million US citizens, and is the most common inherited neuromuscular disorder worldwide. The HNF has made...