by James Radke, PhD | Feb 22, 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 mechanism of action of momelotinib, a JAK-inhibitor being investigated as a treatment for...
by James Radke, PhD | Feb 18, 2022
The U.S. Food and Drug Administration (FDA) has approved mitapivat (Pyrukynd) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. PK deficiency is a rare blood disorder caused by an inherited mutation in the PKLR gene. It is...
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....
