Collaborations play a critical role in advancing potential treatments for a rare disease and can take many forms. In some cases, they may involve a single researcher engaging with a single patient, while more complex ones may be multi-organizational alliances that include drug developers, academic institutions, and patient advocacy organizations. We spoke to Hans Schlecht, a physician who has conducted research and the father of a son with an ultra-rare disease, about the importance of collaborations, how patients can engage researchers, and what they can do to ensure the best returns for their efforts.