Ridgeland, MS — Amicus Therapeutics (FOLD) has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease. The Company has reached agreement with the FDA on the key protocol design elements of the pivotal trial, including the use of the...
Latest News
REDWOOD CITY, Calif. – AmMax Bio, Inc. (“AmMax”), a private clinical-stage biopharmaceutical company developing innovative treatments with its proprietary dual anti-inflammatory/anti-fibrotic therapeutic platform targeting the colony-stimulating factor 1 receptor (CSF1R), today announced that John Varian has been appointed as an Independent Board Member. “We are very happy to welcome John...
– Additional research centers now enrolling patients in Australia and New Zealand – Company has achieved 50 percent of target enrollment in global Phase 3 trial LONDON – AMO Pharma Limited (“AMO Pharma”), a privately held biopharmaceutical company focusing on rare childhood-onset neurogenetic disorders with limited or no treatment options, today announced the...
NEW YORK – Amplo Biotechnology, a privately held US-based biotech focused on developing AAV (Adeno-associated Virus)-based gene therapies for diseases affecting the neuromuscular junction, today announced the successful closing of an undisclosed Series Seed financing. The Seed round was led by BioBrit, LLC., joined by Casdin Capital, SeedFolio and small...
LINKÖPING, Sweden – AMRA announced today that its magnetic resonance imaging (MRI) based method has sufficient sensitivity to detect disease progression at the muscular level in ReDUX4: a multicenter Phase 2b clinical trial testing the safety and efficacy of Losmapimod in treating facioscapulohumeral muscular dystrophy (FSHD). The study introduced a powerful new method in...
ALBANY, N.Y. – Albany Molecular Research, Inc. (AMRI), a leading global provider of advanced contract research, development and manufacturing solutions, today announced expanded access to its accelerated solutions platform, spanning research & development to manufacturing for orphan and rare disease products. The company has made targeted investments globally to expand its...
DUBLIN, Ireland, and Boston MA, December 23, 2020, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, is pleased to announce today that the U.S. Food and Drug Administration (“FDA”) has granted orphan...
Amsterdam, Netherlands – Mycobacterium tuberculosis (Mtb), the bacteria that causes a tuberculosis infection, is present in exhaled breath of 90% of those presenting with suspected tuberculosis. This includes those who were negative on conventional sputum testing and not diagnosed with TB. This raises the possibility that those who have tested...
Amsterdam, Netherlands – Research led by Amsterdam UMC across ten Dutch and two Italian hospitals has found that not placing a drain during surgery improves outcomes in patients undergoing a left-sided pancreatic resection, also known as ‘distal pancreatectomy’. The study, today published in Lancet Gastroenterology & Hepatology, set out to...
AMSTERDAM — Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT’s gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP). Orphan Drug Designation for AIP entitles AMT to...