The Nathan Model is a new model of care we are developing with the help of experts for the management of rare diseases. The model identifies the unmet needs of a single child (in this case, Nathan) across three health domains: clinical and biological, translational, and psychosocial. We then design appropriate research studies to bridge the gaps in knowledge for that child. The aim is to provide a personalised, comprehensive and holistic adjuvant program that can be used to optimize the management of rare diseases, ultimately improving health outcomes and quality of life. It is our hope that this model can one day be used for other children with severe, complex, rare diseases.