TORONTO, Canada — Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that Health Canada has authorized the tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). LIVMARLI had previously been authorized in Canada as an oral solution for the...
treatment News
TORONTO, Canada — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that Health Canada has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies...
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....
OTTAWA, Canada — Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced that Health Canada has granted a marketing authorization for ANDEMBRY® (garadacimab) for routine prevention of attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. ANDEMBRY is a monoclonal antibody designed to target activated FXII (FXIIa),...
Cambridge, UK – Healx, an AI-powered, clinical-stage biotech company dedicated to rare diseases, today announced that the first patient has been dosed in its Phase 1/2 dose-escalation and dose-confirmation clinical trial to evaluate the safety and efficacy of HLX-4310, an investigational oral combination of a class I selective HDAC inhibitor...
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...
CAMBRIDGE, Mass. and COPENHAGEN, Denmark — Hemab Therapeutics (Nasdaq:COAG), a clinical-stage biotechnology company developing therapies that reimagine the treatment of blood coagulation disorders to sustain life and human resilience, today presented clinical and preclinical data from sutacimig in GT and FVIID at the International Society on Thrombosis and Haemostasis (ISTH)...
CAMBRIDGE, Mass. and COPENHAGEN, Denmark — Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced positive results from its completed Phase 2 multiple ascending dose (MAD) portion of the CL-101 study of sutacimig for the prophylactic treatment of Glanzmann thrombasthenia...