Therapy Areas: Hereditary Disorders
8 September 2020 - Biopharmaceutical company Aridis Pharmaceuticals Inc (Nasdaq:ARDS) on Tuesday announced an agreement with the US Food and Drug Administration (FDA) to simplify its AR-501 Phase 2 tr...
4 September 2020 - - US-based orphan lung disease company Savara Inc. (NASDAQ: SVRA) has decided to discontinue ENCORE, a 48-week, open-label, non-controlled Phase 2a exploratory clinical study of Mol...
24 August 2020 - Biotechnology company Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) reported on Friday the receipt of the marketing authorization from the European Commission (EC) for KAFTRIO in ...
18 August 2020 - - UK-based biopharmaceutical company Enterprise Therapeutics Ltd has dosed the first subjects in a Phase 1 trial for its novel inhaled cystic fibrosis therapy, ETD002, the company sai...
13 August 2020 - - The US Food and Drug Administration has granted Fast Track designation to 4D-310 for Fabry disease, a debilitating lysosomal storage disorder caused by a deficiency of alpha-galacto...
12 August 2020 - - US-based Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) has dosed the first subjects in AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC, the company's investigational RNA in...