by Peter Ciszewski | Oct 27, 2020
Eric Jonasch, MD, Professor, Department of Genitourinary Medical Oncology at MD Anderson Cancer Center, gives an overview of von Hippel-Lindau disease. As Dr. Jonasch explains, von Hippel-Lindau disease is a genetic disease characterized by the formation of...
by Peter Ciszewski | Oct 26, 2020
Erik Harris, Executive Vice President and Chief Commercial Officer at Ultragenyx Pharmaceutical Inc. discusses what he has learned being a commercial officer for a rare disease pharmaceutical company. Mr. Harris first notes the uniqueness of each patient’s...
by Peter Ciszewski | Oct 23, 2020
Jim Willson, a patient with spinal muscular atrophy (SMA) who is currently in a clinical trial for risdiplam (Evrysdi), gives advice to physicians and patients thinking about being part of a clinical trial. SMA is a genetic disease that affects motor neurons in...
by Peter Ciszewski | Oct 22, 2020
John Kuruvilla, MD, from the Princess Margaret Cancer Centre provides an overview of treatment options for persons with Hodgkin’s lymphoma. Hodgkin’s lymphoma is a rare cancer that is usually treated initially with salvage therapy and autologous stem cell...
by Peter Ciszewski | Oct 21, 2020
Jim Willison, a patient with spinal muscular atrophy (SMA), discusses his experience being part of a clinical trial for risdiplam (Evrysdi). SMA is a genetic disease that affects motor neurons in the spinal cord and control of muscle movement. It is caused by a...
