Trapping the full-length protein in the ER is also the reason for the 25 kDa size difference between 7 and WT because it cannot obtain the complex glycan modification at the Golgi
NAS, Non-Alcoholic Fatty Liver Activity Score
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Body Weight Change After 52 Weeks of Setmelanotide Treatment Among pivotal patients aged greater than or equal to 12 years with BBS (N = 28), 35.7% (95% confidence interval [CI], 18.6% to 55.9%) had a greater than or equal to 10% reduction in body weight from the active treatment baseline after 52 weeks of treatment with setmelanotide, which was greater than the prespecified assumption of 10% of patients with BBS achieving a greater than or equal to 10% reduction in body weight without setmelanotide treatment
Supplementary Information Additional file 1