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SAY reports acting as a speaker for Chiesi Farmaceutici. RJB has received research support from Chiesi and funding from the Intramural Research Program of the National Institute of Diabetes and Digestive and Kidney Diseases and has also received research support or drug for studies from Regeneron, Marea, and Pfizer. All remaining authors report nothing to disclose.","order":1,"name":"Ethics","label":"Competing Interests","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Approval of this retrospective analysis was obtained from the ethics committee or institutional review board at participating medical centers, as required. This analysis adhered to the tenets of the Declaration of Helsinki [\n                      \n                      ]. Except for two patients, local regulations did not require written consent for the use of retrospective anonymized (unidentifiable) data; however, all patients (or their legal guardians) provided written consent for genetic testing. The remaining two patients previously provided written consent to participate in a prospective, open-label interventional studies of metreleptin conducted at the US National Institute of Health (NIH) Clinical Center, Bethesda, Maryland. Both patients subsequently transitioned to commercial metreleptin and continued to receive medical care at the NIH. Anonymized, retrospective data from both patients were included in the current analysis.","order":2,"name":"Ethics","label":"Ethics approval","group":{"name":"EthicsHeading","label":"Declarations"}}]}}