by James Radke, PhD | Jan 27, 2022
Thomas Martin, MD, UCSF Helen Diller Family Comprehensive Cancer Center, discusses how positive data reflecting the safety and efficacy of CAR T cell therapy to treat multiple myeloma may lead to expanded use of this group of therapies. One example...
by James Radke, PhD | Jan 14, 2022
Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, gives an overview of the treatment landscape for phenylketonuria (PKU), going further into detail about the mechanism of action of SYNB1618, an oral drug...
by James Radke, PhD | Dec 16, 2021
The U.S. Food and Drug Administration (FDA) has approved budesonide (Tarpeyo) to reduce proteinuria in adults with primary immunoglobulin A (IgA) nephropathy. This is the first and only FDA approved drug indicated specifically for the management of IgA nephropathy....
by James Radke, PhD | Dec 13, 2021
Sihoun Hahn, MD, Director of the Wilson Disease Center of Excellence at Seattle Children’s Research Institute, describes the steps necessary for conditions, such as Wilson disease, to be added to the recommended uniform screening panel (RUSP). Wilson disease is...
