DUBLIN – Jazz Pharmaceuticals plc (NASDAQ: JAZZ) today announced the Health Canada approval and availability of Vyxeos® (daunorubicin and cytarabine liposome for injection) for the treatment of adults with newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).[1] AML, a cancer of the bone marrow and the blood, progresses rapidly without treatment.[2]...
treatment News
BOSTON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation....
NEWTOWN, Pa. — Helius Medical Technologies, Inc. (NASDAQ: HSDT) is expanding its presence at the 2024 American Physical Therapy Association (APTA) Combined Sections Meeting (CSM), that will be held February 15 through February 17 at the Boston Convention and Exposition Center: In addition to exhibiting its innovative Portable Neuromodulation Stimulator...
Munich, Germany – Chronic hepatitis B, resulting from infection with the hepatitis B virus (HBV), is an inflammatory liver disease that results in tissue damage. Persistent HBV infection, where the immune system cannot fully eradicate the virus, can lead to continuous liver damage, liver cirrhosis, and even liver cancer. HBV...
COPENHAGEN, Denmark and CAMBRIDGE, Mass. — Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today presented initial results from Phase 1 of its ongoing evaluation of HMB-001. A novel bispecific antibody, HMB-001 is in development as a prophylactic treatment for the...
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...
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...
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...