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®), a liquid medication that supplies medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with long-chain fatty acid oxidation disorder (LCFAOD).

Read the full article: Improving the Lives of Patients With LCFAOD


Continue Reading