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...
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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...
CAMBRIDGE, Mass.— Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced interim data from the ongoing Phase 2 HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with Wolfram syndrome, a rare, progressive genetic disease impacting approximately 3,000 people in the U.S. The...
CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced it has completed enrollment of its Phase 2 HELIOS trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS), a rare genetic disease that typically results in neurodegeneration and premature death, which has no...
CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. today announced that the first participant has been dosed in ORION, a randomized, double-blind, placebo-controlled Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of progressive supranuclear palsy (PSP). The Phase 3 trial will enroll approximately 600 participants...
CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc. today announced that the first participants have been dosed in the Phase 3 Study A35-004 (PHOENIX) (NCT05021536), a global trial evaluating the safety and efficacy of AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine)) in people with amyotrophic lateral sclerosis (ALS)....