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Medication Delivery : Once approved, your medication will be sent to you directly, with clear instructions for use

Abbreviations AE adverse event AS Alstrm syndrome BBS Bardet-Biedl syndrome BMI body mass index CI confidence interval CRIBBS Clinical Registry Investigating Bardet-Biedl Syndrome FAS full analysis set GLP1 glucagon-like peptide 1 HRQoL health-related quality of life IWQoL-Lite Impact of Weight on Quality of Life Lite LEPR leptin receptor LTE long-term extension MC4R melanocortin-4 receptor MID minimally important difference PCSK1 proprotein convertase subtilisin/kexin type 1 PedsQL Pediatric Quality of Life Inventory POMC pro-opiomelanocortin PRO patient-reported outcome QoL quality of life RCT randomized controlled trial SAE serious adverse event SAS safety analysis set SC subcutaneous SD standard deviation TEAE treatment-emergent adverse event Appendix 1

Patients may need 100500 mg/day IV thiamine to prevent or sometime leads Wernickes encephalopathy a life-threatening complication causing confusion, memory loss, and poor coordination

Based on your health profile, they may decide that theyre appropriate if the benefits outweigh the risks

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