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...

Paris, France – The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the treatment of neurological manifestations of Gaucher disease type 3 (GD3), a rare lysosomal storage disorder. The designation is based on data from the LEAP2MONO...

SAN FRANCISCO, CA – Jaguar Health, Inc. (NASDAQ:JAGX) family company Napo Pharmaceuticals (Napo) today announced that, based on the U.S. Food and Drug Administration (FDA) support for Napo’s protocol amendment for its clinical trial to evaluate the safety and efficacy of its novel crofelemer powder-for-oral-solution formulation in pediatric patients with intestinal...