treatment News

MELBOURNE, Australia – Neuren Pharmaceuticals Limited (ASX: NEU) has received clear and constructive guidance from the FDA Office of Neuroscience in pre-IND meetings for Phase 2 clinical trials of NNZ-2591 in Phelan-McDermid, Angelman and Pitt Hopkins syndromes. All three syndromes are serious neurological disorders that emerge in early childhood and have no...
Data demonstrates the potential for INCA033989 to modify disease by directly inhibiting and eliminating oncogenic mutCALR cells, while sparing healthy cells and restoring normal blood cell production In high-risk patients with essential thrombocythemia (ET) with a CALR mutation (mutCALR), 86% of INCA033989-treated patients at doses 400 mg and above achieved...
Announcing the results of the TALENTACE study for unresectable hepatocellular carcinoma eligible for transarterial chemoembolization (TACE) without prior systemic treatment The study demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of TACE-progression-free survival (TACE PFS), and overall survival (OS) is immature at the prespecified first interim...
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...
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...