STOCKHOLM – Swedish Orphan Biovitrum AB (publ) (Sobi™) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) (GLOBE NEWSWIRE) today reported positive top-line results from the phase 3 PRINCE study evaluating the efficacy and safety of pegcetacoplan in adults with paroxysmal nocturnal haemoglobinuria (PNH) who are treatment naïve, meaning they had not received a...
treatment News
ATLANTA — UCB, a global biopharmaceutical company, will present a post hoc analysis of the EXXELERATE trial examining the efficacy of CIMZIA® (certolizumab pegol) and adalimumab in patients with rheumatoid arthritis (RA) with high rheumatoid factor (RF) levels. The data are being presented at the American College of Rheumatology (ACR)...
JERSEY CITY, N.J. – Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced a post-hoc analysis of its Phase 3 edaravone study reviewing the results of intravenous (IV) edaravone treatment on disease progression milestones and events among people with amyotrophic lateral sclerosis (ALS). In the analysis, a risk reduction was observed...
HAIFA, Israel and MIAMI, FL — Insightec, a global healthcare company dedicated to using acoustic energy to transform patient care, today announced a promising clinical study in the treatment of Alzheimer’s disease leveraging focused ultrasound (FUS) technology, which has been reported in the latest issue of The New England Journal...
Pittsburgh, PA – In a Nature Metabolism paper published today, researchers from the University of Pittsburgh detail a potential new target and a small-molecule drug candidate for treating Barth syndrome, a rare, life-threatening and currently incurable genetic disease with devastating consequences. Barth syndrome affects about 1 in every 300,000 to...
Utrecht, Netherlands – The Organoid Group (Hubrecht Institute) and the Rare Cancers Genomics Team (IARC/WHO) found a way to grow samples of different types of neuroendocrine tumors (NETs) in the lab. While generating their new model, the researchers discovered that some pulmonary NETs need the protein EGF to be able...
NASHVILLE, Tenn. – A potential schizophrenia treatment discovered through the Warren Center for Neuroscience Drug Discovery has been cleared by the U.S. Food and Drug Administration for use in phase 1 clinical trials—the third WCNDD therapeutic to reach that benchmark. The drug is an allosteric modulator that works through a...
BIRMINGHAM, Ala. – It has been uncertain how Mycobacterium tuberculosis deflects the immune response in humans, though evidence has pointed to host immunometabolism — the intrinsic link between metabolism in immune cells and their immune function. The pathogen M. tuberculosis is known to disrupt a metabolic pathway called glycolysis in...
BOSTON, Mass. — Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced positive topline results from the EMBRAVE Part A trial of elsunersen in pediatric patients with early-seizure onset SCN2A developmental and epileptic encephalopathy (DEE). “We are thrilled to see...
BOSTON, Mass. — Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that, following a comprehensive Type B meeting and recent meeting minutes and written advice on the...