by Peter Ciszewski | Apr 13, 2021
Stacy Pokorny, a mother of a young girl with spinal muscular atrophy (SMA), gives advice to parents of children who have recently been diagnosed with the disease. SMA is a rare inherited neuromuscular disorder caused by an inadequate level of the survivor...
by Peter Ciszewski | Apr 12, 2021
Rudolf Baumgartner, MD, Chief Medical Officer and Head of Clinical Development at Saniona, gives an overview of Prader-Willi syndrome (PWS) and tesomet, a drug combination under investigation for the treatment of PWS. As Dr. Baumgartner explains, PWS is a rare...
by Peter Ciszewski | Apr 12, 2021
Jonathan Hill, a patient with hemophilia A, talks about the many challenges faced by the hemophilia community during the last part of the twentieth century. Hemophilia A is a genetic disorder caused by missing or defective clotting protein, factor VIII....
by Peter Ciszewski | Apr 12, 2021
Shruti Chaturvedi, MBBS, MS, from Johns Hopkins and X. Long Zheng, MD, PhD, from the University of Kansas Medical Center, discuss how to manage thrombotic thrombocytopenic purpura (TTP) during pregnancy. TTP is a rare hematologic condition that...
by Peter Ciszewski | Apr 9, 2021
Florence Wong, MD, Hepatologist at Toronto General Hospital discusses terlipressin, a potent vasopressin analogue being investigated for the treatment of hepatorenal syndrome type 1 (HRS-1). In the first part of the interview, Dr. Wong describes the...
