by Peter Ciszewski | Jun 20, 2018
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of his company’s leading drug candidate, ELX-02 for cystic fibrosis. ELX-02 is designed to increase the read-through activity in...
by Peter Ciszewski | Jun 20, 2018
Alexion Pharmaceuticals has submitted a Biologics License Application (BLA) to the YOU.S. Food and Drug Administration (FDA) for approval of ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, potentially deadly...
by Peter Ciszewski | Jun 18, 2018
BioMarin Pharmaceutical dosed the first participant in their global phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in infants and young children with achondroplasia. Achondroplasia is a skeletal dysplasia and is characterized by the...
by Peter Ciszewski | Jun 16, 2018
The U.S. Food and Drug Administration has granted Orphan Drug Designation to AMO Pharma for it’s investigational therapy called AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder. “This designation is an important...
by Peter Ciszewski | Jun 15, 2018
Rhythm Pharmaceuticals announced that it has completed enrollment of the pivotal cohorts of 10 patients in two separate, ongoing, registration-enabling Phase 3 clinical trials evaluating setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR)...
