Aug 31, 2021
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Peter Knox, senior research director at the Life Raft Group. Based in Wayne, New Jersey, this organization was incorporated in 2002 and advocates for people with all forms of gastrointestinal stromal tumor, or...
Aug 17, 2021
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Mark Brantly, MD, scientific director of the Alpha-1 Foundation, ahead of the organization's August 28, 2021, "Virtual Education Day." Dr. Brantly is the vice-chair of research in the Department of Medicine at the University of Florida in...
Aug 11, 2021
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Camille Bedrosian, MD, chief medical officer and executive vice president of Ultragenyx Pharmaceutical in Novato, California. Earlier this year, Ulragenyx won approval from the US Food and Drug Administration (FDA) for triheptanoin (Dojolvi®),...