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...
treatment News
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)....
CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc. today announced its intention to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO)) for the treatment of amyotrophic lateral sclerosis (ALS). The decision by the company to submit the application in...
CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced plans to submit a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in the first half of...
CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced that two abstracts detailing next steps in the development of the Company’s investigational antisense oligonucleotide (ASO), AMX0114, for the potential treatment of amyotrophic lateral sclerosis (ALS) and findings from a pharmacokinetic and pharmacodynamic study of AMX0035 (sodium phenylbutyrate [PB] and...
HCC is the fifth most common cancer in the world and the third most frequent cause of cancer-related death. Only about 20% of HCC patients are eligible for surgical resection. Therefore, RFA has been used increasingly as a safe technique for treating hepatic tumors. However, for hypervascular HCC, RFA appears...