Mikaella Caruncho

Mar 21, 2023 | gimjournal.org | Sandra Lee |Wendy Chung |Josephine Johnston |Mikaella Caruncho

Advances in the study of ultra-rare genetic conditions are leading to the development of targeted interventions developed for single or very small numbers of patients. Due to the experimental but also highly individualized nature of these interventions, they are difficult to classify cleanly as either research or clinical care.