by Peter Ciszewski | May 16, 2018
Albireo Pharma announced that the first patient has been enrolled, a Phase 3 clinical trial (called PEDFIC-1) for patients with progressive familial intrahepatic cholestasis (PFIC), a rare and progressive liver disease, which typically leads to liver failure. In...
by Peter Ciszewski | May 14, 2018
Pulmonary vein stenosis (PVS) is a rare disease in which abnormal cells build up inside the veins responsible for carrying oxygen-rich blood from the lungs to the heart. It restricts blood flow through these vessels, eventually sealing them off entirely if left...
by Peter Ciszewski | May 12, 2018
Yuval Cohen, PhD, CEO of Corbus provides and overview of systemic sclerosis (SSc), a rare and serious systemic autoimmune rheumatic disease with an unclear etiology. Systemic sclerosis affects approximately 90,000 people in the United States and Europe, with...
by Peter Ciszewski | May 11, 2018
Dr. John Maraganore, CEO of Alnylam discusses Henri A. Termeer Tribute Committee. A number of industry leaders have joined forces to form this Committee with the mission to commemorate Henri Termeer’s remarkable stewardship of the local biotech industry and his...
by Peter Ciszewski | May 11, 2018
An FDA advisory committee voted to recommend the approval of volanesorsen (Waylivra), an RNAi drug for rare lipid disorder familial chylomicronemia syndrome (FCS). The FDA is expected to make it’s final decision by Aug. 30. The Endocrinologic and Metabolic Drugs...
