ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Lancet. 2019 Nov 30;394(10213):2012-2024. doi: 10.1016/S0140-6736(19)32517-6.Resmetirom (MGL-3196) is a liver-directed, orally active, selective thyroid hormone receptor-β agonist. This 36-week long randomised, placebo-controlled trial in 348 US adults with biopsy confirmed non-alcoholic steatohepatitis (fibrosis stages 1–3) shows that Resmetirom...

Homozygous familial hypercholesterolemia (HoFH) is a severe disorder caused by genetic mutations in LDLR (encoding the LDL receptor), APOB or PCSK9. LDL-C levels in HoFH are extremely elevated)>400 mg/dL(even in utero, leading to cardiovascular events, and disability or death during childhood and adolescence. Conventional medications have minimal efficacy, since LDL-C levels cannot be reduced through upregulation of hepatic LDL receptors.<p c...

In Brief This phase 3 trial including 966 adults with biopsy-confirmed non-alcoholic steatohepatitis (NASH) with fibrosis were randomly assigned (1:1:1 ratio) to once-daily resmetirom 80 mg or 100 mg or placebo. Resmetirom, both 80 and 100 mg daily, were superior to placebo with respect to NASH resolution and improvement in liver fibrosis.NASH resolution occurred more frequently in patients on 80-mg (25.9%) and 100-mg resmetirom (29.9%) than on placebo (...