by Peter Ciszewski | Feb 20, 2019
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses Primary Hyperoxaluria Type 1 and his company’s investigational RNAi therapy, lumasiran. Primary hyperoxaluria type 1 (PH1) is a rare disorder that mainly affects the kidneys. It results from...
by Peter Ciszewski | Feb 19, 2019
Manuel Litchman, MD, President, Chief Executive Officer at Mustang Bio discusses his company’s clinical program focused on X-linked severe combined immunodeficiency (SCID). X-linked SCID is an inherited disorder of the immune system that occurs almost...
by Peter Ciszewski | Feb 18, 2019
Armando Anido, Chief Executive Officer at Zynerba Pharmaceuticals discusses Fragile X syndrome, a genetic condition involving changes in part of the X chromosome. This condition causes a range of developmental problems including learning disabilities and...
by Peter Ciszewski | Feb 17, 2019
February 28 is the 12th Annual Rare Disease Day. This year’s theme is “bridging health and social care,” and will bring together the rare disease community in various cities across the world, highlighting the need for better medical, social, and support services...
by Peter Ciszewski | Feb 15, 2019
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. Catalyst is a clinical-stage biopharmaceutical company developing novel medicines to...
