by James Radke, PhD | Feb 16, 2022
Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses the phase 1/2 trial of the gene therapy, 4D-310, in Fabry disease. Data from this study was recently presented at WORLDSymposium 2022. Fabry...
by James Radke, PhD | Feb 8, 2022
Jeffrey Neul, MD, PhD, Professor of Pediatrics at Vanderbilt University Medical Center, discusses recent top-line results from a phase 3 trial testing trofinetide to treat children with Rett syndrome. Trofinetide is an analog of insulin-like growth factor 1...
by James Radke, PhD | Feb 2, 2022
Srdan Verstovsek, MD, PhD, Medical Oncologist and Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, discusses the top-line results from the phase 3 MOMENTUM trial of momelotinib in myelofibrosis patients....
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...
