by Peter Ciszewski | Jun 21, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. This condition affects many different parts of the body and occurs almost...
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...
