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., 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...
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...
Anacor Pharmaceuticals, a biopharmaceutical company engaged in the development of small molecule drugs, has entered into a licensing and research agreement with not-for-profit Global Alliance for TB Drug Development or TB Alliance to explore a novel anti-bacterial drug target for use in tuberculosis therapy. Under the agreement, Anacor will provide...
NEW YORK – Anavex Life Sciences Corp. (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that the Independent Data Safety Monitoring Board (DSMB) for the...
BRIDGEWATER, N.J. – Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved ARIKAYCE®(amikacin liposome inhalation suspension) for the treatment of patients with nontuberculous mycobacterial (NTM)...
Cardiac amyloidosis is a rare disease characterized by the accumulation of plaques of the amyloid protein in the heart, making it hard (which is why it is also called stiff heart syndrome) and may require a pacemaker or be the cause of death. It has some treatments, but they are...
SOUTH SAN FRANCISCO, Calif.- Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, hematologic cancers and autoimmune disease, today announced a combined long-term overall survival (OS) analysis from three clinical...
Audentes Therapeutics (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced positive interim data from the first dose cohort of ASPIRO, a Phase 1/2 clinical trial of AT132 in patients with X-Linked Myotubular Myopathy (XLMTM). ASPIRO is a...