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 ...

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...

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-...

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 peopl...