by Peter Ciszewski | Oct 8, 2020
Erik Harris, Executive Vice President and Chief Commercial Officer at Ultragenyx Pharmaceutical Inc. discusses the recent approval of triheptanoin (Dojolvi) and gives an overview of long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD is a group of...
by Peter Ciszewski | Oct 7, 2020
Mary Smith, PhD of SpringWorks Therapeutics explains the pathophysiology and diagnosis of Desmoid tumors. Desmoid tumors are benign soft tissue tumors that can invade surrounding healthy tissues, including joints, muscle, and viscera. These rare tumors can arise...
by Peter Ciszewski | Oct 5, 2020
Tiffany Cook, Senior Director of CureDuchenne, discusses the upcoming Duchenne United Gaming Twitch Tournament on October 10, 2020, 12 p.m. EST. Duchenne muscular dystrophy is the most common and most severe form of muscular dystrophy, caused by loss of...
by Peter Ciszewski | Oct 2, 2020
Emil Kakkis, MD, PhD, President and Founder of Ultragenyx Pharmaceutical Inc. and EveryLife Foundation, discusses the role patient groups play in the rare disease drug development process. According to Dr. Kakkis, patient groups often provide support for...
by Peter Ciszewski | Oct 2, 2020
Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD), discusses the upcoming Virtual Rare Summit being held October 8-9. For over 30 years, NORD has been providing services for rare disease patients and...
