ESPE Abstracts

ESPE Abstracts

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hrp0098p1-296 | Late Breaking 1 | ESPE2024

Evaluating Setmelanotide Treatment for 12 Months in Pediatric Age Groups With Rare Melanocortin-4 Receptor Pathway–Related Obesity: Efficacy in Weight Reduction and Safety Outcomes

Kühnen Peter , L. T. van den Akker Erica , H. Shoemaker Ashley , Okorie Uzoma , F. Verge Charles , Fennoy Ilene , M. Kelsey Megan , M. Haqq Andrea , L. Roth Christian , C. Garrison Jill , Wabitsch Martin , Farooqi Sadaf , Argente Jesús

Objectives: Hyperphagia and severe obesity may result from impaired melanocortin-4 receptor (MC4R) signaling due to rare biallelic variants in POMC or PCSK1 (proopiomelanocortin [POMC] deficiency) or LEPR (leptin receptor [LEPR] deficiency), Bardet-Biedl syndrome (BBS) or acquired hypothalamic obesity (HO). Previously, setmelanotide in patients aged 2-17 years was well tolerated and improved weight-related measures and hunger severit...
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hrp0098fc6.4 | Fat, Metabolism and Obesity 1 | ESPE2024

Relaxation of Food Control Parameters Based on Improvements in the Food Safe Zone Questionnaire Occurs with Reduction of Hyperphagia in Clinical Trials of Diazoxide Choline Extended Release (DCCR) in Participants with Prader-Willi Syndrome

Bridges Nicola , Gevers Evelien , Yanovski Jack , Salehi Parisa , Hall Shoemaker Ashley , Abuzzahab Jennifer , Obrynba Kathryn , Fleischman Amy , Stevenson David , Wilding John , Mathew Verghese , Viskochil David , Felner Eric , E. McCandless Shawn , Bird Lynne , Lah Melissa , Littlejohn Elizabeth , Shea Heidi , Holland Anthony , Goldstone Anthony , Barrett Timothy , Konczal Laura , Dharmaraj Poonam , Das Urmi , Angulo Moris , Kimonis Virginia , Guftar Shaikh Mohomad , Mejia-Corletto Jorge , Yen Kristen , Gandhi Raj , Ballal Shaila , Hirano Patricia , Cowen Neil , Bhatnagar Anish , Huang Michael , Butler Merlin , Miller Jennifer

Background: Prader-Willi syndrome (PWS) is a rare genetic neurobehavioral-metabolic disorder characterized by hyperphagia and behavioral/psychological complications. With no approved therapies to treat hyperphagia, disease management requires strict dietary and environmental controls to restrict access to food. DCCR is an oral, once-daily medication currently under development for the treatment of PWS.Objective: The obje...
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hrp0098fc11.4 | Fat, Metabolism and Obesity 2 | ESPE2024

Long-term Efficacy Results of Diazoxide Choline Extended-Release (DCCR) Tablets in Participants with Prader-Willi Syndrome from the Completed C601 (DESTINY PWS) and C602 Open Label Extension (OLE) Studies

Gevers Evelien , Bridges Nicola , Yanovski Jack , Felner Eric , Salehi Parisa , Hall Shoemaker Ashley , Fleischman Amy , Goldstone Anthony , Angulo Moris , Stevenson David , Obrynba Kathryn , Guftar Shaikh M , Holland Anthony , Mathew Verghese , Viskochil David , E. McCandless Shawn , Bird Lynne , Lah Melissa , Kimonis Virginia , Abuzzahab Jennifer , Konczal Laura , Littlejohn Elizabeth , Shea Heidi , Dharmaraj Poonam , Das Urmi , Mejia-Corletto Jorge , Wilding John , Harwood Katerina , Yen Kristen , Gandhi Raj , Gong Jing , Hirano Patricia , Cowen Neil , Bhatnagar Anish , Huang Michael , Butler Merlin , Miller Jennifer

Background: Prader-Willi syndrome (PWS) is a rare genetic neurobehavioral-metabolic disorder characterized by hyperphagia and behavioral/psychological complications. No approved therapies exist for treating hyperphagia in patients with PWS. DCCR is an oral, once-daily medication currently under development for the treatment of PWS.Objective: The objective was to determine the efficacy of investigational DCCR on hyperphag...
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hrp0089rfc11.1 | Bone, Growth Plate & Mineral Metabolism 2 | ESPE2018

Diagnosis and Management of Pseudohypoparathyroidism and Related Disorders: First International Consensus Statement

Mantovani Giovanna , Bastepe Murat , Monk David , de Sanctis Luisa , Thiele Susanne , Usardi Alessia , Ahmed Faisal , Bufo Roberto , Choplin Timothee , DeFillipo Gianpaolo , Devernois Guillemette , Eggermann Thomas , Elli Francesca M , Freson Kathleen , Ramirez Aurora Garcia , Germain-Lee Emily , Groussin Lionel , Hamdy Neveen , Hanna Patrick , Hiort Olaf , Juppner Harald , Kamenicky Peter , Knight Nina , Kottler Marie-Laure , Le Norcy Elvire , Lecumberri Beatriz , Levine Michael A , Makiti Outi , Martin Regina , Martos-Moreno Gabriel Angel , Minagawa Masanori , Muray Philip , Pereda Arrate , Pignolo Roberto , Rejnmark Lars , Rodado Rebecca , Rothenbuhler Anya , Saraff Vrinda , Shoemaker Ashley , Shore Eileen M. , Silve Caroline , Turan Serap , Woods Philip , Zillikens M. Carola , de Nanclares Guiomar Perez , Linglart Agnes

Pseudohypoparathyroidism (PHP) and related disorders lead to a wide spectrum of abnormal physical characteristics, neurocognitive and endocrine abnormalities. PHP (including all subtypes), pseudoPHP, acrodysostosis and progressive osseous heteroplasia refer to heterogeneous disorders characterized by physical findings, differently associated in each subtype, including short bones, short stature, stocky build, subcutaneous ectopic ossifications, as well as laboratory abnormalit...
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