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)...
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Tempe, AZ – The human brain is an incredibly adaptable organ, often able to heal itself even from significant trauma. Yet for the first time, new research shows even brief contact with a common herbicide can cause lasting damage to the brain, which may persist long after direct exposure ends....
Watertown, Mass. — Arkuda Therapeutics, a biotechnology company applying novel insights at the intersection of lysosomal biology and neuronal health to develop medicines that change the trajectory of neurodegenerative disease, announced Arkuda co-founder and previous Senior Vice President, Head of Discovery, Duane Burnett, PhD, has been appointed Chief Technology Officer,...
COLUMBUS, Ohio — Armatus Bio, a late-preclinical stage biotech innovator developing vectorized RNAi medicines in neuromuscular disorders, today announced the publication of foundational data in Molecular Therapy Nucleic Acids describing the safety and biodistribution impact of TVR110, a first-in-class and potential best-in-class vectorized microRNA in development for Charcot-Marie-Tooth disease type 1A...
ARDSLEY, N.Y. – ARMGO Pharma Inc., a clinical stage biopharmaceutical company advancing a novel class of small molecule drugs known as Rycals®, announced today the publication of the results from a Phase 1b study of its Rycal ARM210 (also known as S48168), for the treatment of Ryanodine Receptor 1 Related...
Devon Parks doesn’t just believe in miracles — she says she has proof they exist. Parks’ 3-year-old son, Isaiah, was born with the rare disease called DiGeorge Syndrome, but he has outlived his 2-year life expectancy. He recently was named 2009 Child of the Year by the Pennsylvania Elks Home...
PHILADELPHIA, PA — Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced that it has completed enrollment in its ongoing Phase 1b clinical trial of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD). The trial is evaluating safety,...
PHILADELPHIA, PA — Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines in areas of high unmet need in rare muscle diseases, immunology, and oncology, today announced positive topline results from the Phase 1b portion of its study evaluating ABX1100, a novel investigational therapy for...
WALTHAM, Mass. — Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1),...
PASADENA, Calif. – Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced ARO-DUX4 as Arrowhead’s first muscle targeted investigational RNAi therapeutic candidate to utilize its proprietary Targeted RNAi Molecule (TRIMTM) platform. ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients...