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ey0020.3-2 | Novel Treatments for Rare Skeletal Disorders | ESPEYB20

3.2. Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamics results of a long-acting C-type natriuretic peptide prodrug, TransCon CNP

VM Breinholt , PH Mygind , ED Christoffersen , Y Zhang , S Ota , R Will Charlton , D Viuff

In Brief: This phase 1 study in healthy adults assessed the safety and feasibility of transcon-CNP, a novel prodrug that releases native C-type natriuretic peptide (CNP). The novel drug was well tolerated and CNP remained in systemic circulation for >7 days following a single dose.Commentary: Achondroplasia is caused by autosomal activating mutation in the fibroblast growth factor receptor 3 gene (FGFR3) resulting in constitutive receptor ac...

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ey0019.4-8 | Long-acting growth hormone (LAGH) | ESPEYB19

4.8. Weekly lonapegsomatropin in treatment-naive children with growth hormone deficiency: the phase 3 height trial

PS Thornton , AK Maniatis , E Aghajanova , E Chertok , E Vlachopapadopoulou , Z Lin , W Song , E Dam Christoffersen , VM Breinholt , T Kovalenko , E Giorgadze , M Korpal-Szczyrska , PL Hofman , DB Karpf , AD Shu , Beckert M. Michael

J Clin Endocrinol Metab, 2021. 106(11): p. 3184-3195. PMID: 34272849Brief Summary: This randomized, open-label, controlled, phase 3 trial compared the effects of once-weekly long-acting GH lonapegsomatropin versus daily GH in GHD children over a period of 52 weeks. Efficacy was evaluated by height velocity (HV) and height gain from baseline to end. The long-acting formulation showed not only non-inferiority but also superior efficacy compared to...

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ESPE Yearbook of Paediatric Endocrinology

3.2. Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamics results of a long-acting C-type natriuretic peptide prodrug, TransCon CNP

4.8. Weekly lonapegsomatropin in treatment-naive children with growth hormone deficiency: the phase 3 height trial

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