I am BIO Podcast

There are over 7000 rare diseases identified in the U.S. alone. Many of these diseases affect children, leaving families desperate for answers. In this episode, we talk with one of those families and the biotech company offering them new hope. We also talk with an expert on policy that has jump-started R&D for rare diseases.

David Fajgenbaum was in his third year of medical school when he was diagnosed with a rare and life-threatening disease that began shutting down his organs, bringing him perilously close to death. Although he survived the initial episode, he faced four additional relapses, each pushing him to the brink of death. In this episode, we speak with David about his relentless journey to discover the treatment that ultimately put his disease into remission. We also explore how his personal battle inspired the creation of a groundbreaking approach to help others suffering from rare diseases.

Bradley Burnam woke up one morning, looked in the mirror and found one ear twice it’s normal size and his face swollen and discolored. He would spend the next several years in and out of the hospital fighting a relentless infection that would not respond to available treatments. The experience took him from patient to "mad scientist" to biotech company founder. In this episode, Bradley takes us through his desperate journey to find a cure. We also talk with the leader of an organization helping to get more antimicrobials to the marketplace.

When people were dying from AIDS in the 1980s and 90s, Congress found a way to expedite the drug approval process and saved countless lives. Established in 1992, the Accelerated Approval Program allows the FDA to speed approval of drugs for patients with serious and life-threatening conditions. In this episode we talk with the father of children with a rare disease, a company CEO working to treat rare diseases and two policy experts on Accelerated Approval.

Patient Advocates do more than raise money—they are active participants in the drug development process. While the ALS Ice Bucket Challenge garnered global attention, lesser-known patient advocates also flex their expertise to drive new treatments and cures.  In this episode, we talk with two moms, one who is a patient herself, about their work as partners in research and development, helping bring treatments for two rare diseases to the market.