by Peter Ciszewski | Mar 8, 2020
The U.S. Food and Drug Administration (FDA) has approved Isturisa (osilodrostat) to treat adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. Cushing’s disease is a rare endocrine...
by Peter Ciszewski | Mar 4, 2020
James Dean, MD, Senior Medical Director at Pharmacyclics provides an update on the phase II CAPTIVATE study testing ibrutinib plus venetoclax to treat chronic lymphocytic leukemia (CLL) or small lymphocytic leukemia (SLL). CLL is a rare blood cancer resulting...
by Peter Ciszewski | Mar 3, 2020
David Kronn, MD, Associate Professor of Pediatrics at New York Medical College, describes the latest results from the mini-COMET phase II study testing avalglucosidase alfa to treat Pompe disease. Avalglucosidase alfa is a second-generation enzyme replacement therapy...
by Peter Ciszewski | Mar 2, 2020
The U.S. Food and Drug Administration (FDA) has approved Sarclisa (isatuximab-irfc), in combination with pomalidomide and dexamethasone, to treat adult patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a...
by Peter Ciszewski | Mar 2, 2020
Barbara Burton, MD of Lurie Children’s Hospital of Chicago explains Morquio syndrome type A, a rare lysosomal storage disorder. Morquio syndrome type A, or mucopolysaccharidosis (MPS) IVA, is due to a mutation in the GALNS gene that leads to a deficiency in the...
