by Peter Ciszewski | Feb 28, 2022
Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, gives an update on the phase 2 trial testing SYNB1618 to treat phenylketonuria (PKU). PKU is a rare genetic metabolic disorder that results in reduced...
by Peter Ciszewski | Feb 24, 2022
Roberto Giugliani, MD, PhD, Professor at the Department of Genetics of the Federal University of Rio Grande do Sul (UFRGS), Chief of the Medical Genetics Center of Hospital de Clinicas de Porto Alegre, and Director of the WHO Collaborating Centre for the...
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 CheckRare Staff | Feb 21, 2022
Stefan M. Schieke, MD, Assistant Professor of Dermatology at University of Wisconsin-Madison discusses cutaneous T-cell lymphoma (CTCL) in special populations such as African Americans and veterans of war. According to a paper published in U.S. Medicine, “The...
by Peter Ciszewski | Feb 21, 2022
Brahm Goldstein, MD, Global Therapeutic Area Head of Hematology and Thrombosis at CSL Behring, gives an overview of hemophilia B. As Dr. Goldstein explains, hemophilia B is a congenital bleeding disorder due to dysfunction or deficiency of coagulation Factor IX...
