The Zebras Herd

Allyson Berent is the Chief Scientific Officer of the Foundation for Angelman Syndrome Therapeutics (FAST). A veterinarian by training, Allyson has been instrumental in the progress that has been made towards the developments of treatments for Angelman Syndrome. In this wide-ranging conversation, she shares with us the keys to the success of FAST, as well as the ways in which she is trying to help other disease foundations by sharing her experience.

Tim Yu is a neurologist at Boston Children’s Hospital. He is responsible for one of the first examples of individualized genetic medicine – the drug milasen. In this episode, we spoke about his approach to drug development, about how he engages with ultrarare patients, about his thoughts on equity, and about the impact of the N=1 Collaborative.

Terry Pirovolakis, CEO of Elpida Therapeutics, achieved what many parents in the ultrarare-disease community aspire to: developing a therapy for his son. He sat down with us to discuss the lessons that he learned as he navigated the whole drug-discovery process in less than two years, laying out the foundation for what now is Elpida.

Steven Gray is Associate Professor at UTSW. He is a world leader in gene therapy for ultrarare diseases. During this wide-ranging conversation, we spoke about the keys to his success, how he engages with disease foundations, how he trains people, and the future of the field.