by James Radke, PhD | Feb 21, 2023
Allen Davidoff, PhD, CEO of XORTX Therapeutics, gives an overview of autosomal dominant polycystic kidney disease (ADPKD). As Dr. Davidoff explains, ADPKD is a genetic disorder characterized by the formation of cysts in the kidneys. The most common symptoms –...
by James Radke, PhD | Feb 17, 2023
The U.S. Food and Drug Administration has approved velmanase alfa-tycv for the treatment of non-central nervous system manifestations of alpha-mannosidosis adult and pediatric patients. Alpha mannosidosis is a multisystemic, lysosomal disorder caused by a deficiency...
by James Radke, PhD | Jan 29, 2023
The U.S. Food and Drug Administration (FDA) has approved zanubrutinib (Brukinsa) to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). CLL is a rare blood cancer resulting in a build-up of lymphocytes in bone marrow, lymph nodes,...
by James Radke, PhD | Jan 4, 2023
Adeline Vanderver, MD, Program Director of the Leukodystrophy Center in the Division of Neurology at Children’s Hospital of Philadelphia (CHOP), Professor of Neurology and Pediatrics, Perelman School of Medicine of the University of Pennsylvania, and Jacob A....
by James Radke, PhD | Dec 16, 2022
Alan M. Mendelsohn, MD, Chief Medical Officer at Timber Pharmaceuticals, provides an overview of congenital ichthyosis. As Dr. Mendelsohn explains, congenital ichthyosis is a rare genetic skin disorder. It is characterized by dry, thickened, and scaling...
