Nassim Usman, PhD, President and Chief Executive Officer of Catalyst Biosciences provides an overview of his company and its focus on protease-based therapeutic candidates.
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses Acute Hepatic Porphyria (AHPs) and givosiran, an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of AHP.
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses hereditary ATTR (hATTR) amyloidosis, a rare condition that affects an estimated 50,000 people worldwide.
Alexion announced positive top line results in the Phase 3 study of Ultomiris (ravulizumab-cwvz), the long-acting C5 complement inhibitor, met its primary objective in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome (aHUS).
Brian Schwartz, Chief Medical Officer of ArQule, discusses his company focus on Proteus syndrome, a rare condition characterized by overgrowth of the bones, skin, and other tissues.
Rod Humerickhouse, MD, PhD, asset strategy leader at AbbVie discusses the potential use of venetoclax for the treatment of patients with multiple myeloma at the 60th ASH Annual Meeting in San Diego.