ESPE Abstracts

ESPE Abstracts

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hrp0097p1-343 | Pituitary, Neuroendocrinology and Puberty | ESPE2023

Facilitating Transition of Care Into Adulthood in Brain Cancer Survivors With Acquired Pediatric Growth Hormone Deficiency: Insights From an Advisory Board

Alter Craig , Boguszewski Margaret , Clemmons David , Dobri Georgiana , Geffner Mitchell , Kelepouris Nicky , Miller Bradley , Oh Rich , Shea Heidi , Yuen

Kevin

Childhood cancer survivors (CCS), particularly brain cancer survivors, are at risk of developing growth hormone deficiency (GHD) due to hypothalamic-pituitary damage from direct tumor mass effects or treatment. Optimization of testing, long-term treatment, and monitoring during care transition from pediatric to adult endocrinology providers remain challenging. A group of endocrinology experts convened to discuss these challenges, the risks and benefits of GH therapy in CCS wit...
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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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