Comparing the efficacy and safety of weekly somatrogon with daily somatropin to treat children with growth hormone deficiency: a plain language summary of publication

Autor: Cheri L. Deal, Joel Steelman, Elpis Vlachopapadopoulou, Renata Stawerska, Lawrence A. Silverman, Moshe Phillip, Ho-Seong Kim, CheolWoo Ko, Oleg Malievskiy, Jose F. Cara, Carl L. Roland, Carrie Turich Taylor, Srinivas Rao Valluri, Michael P. Wajnrajch, Aleksandra Pastrak, Bradley S. Miller

Publikováno v: Therapeutic Advances in Endocrinology and Metabolism, Vol 15 (2024)

• The efficacy of weekly somatrogon injections was no different from that of daily somatropin injections to treat children who don’t make enough growth hormone to grow adequately. ○ Efficacy refers to how well a drug works in a clinical trial.

Externí odkaz: https://doaj.org/article/775d02973cf444caab3d2ae30fd5b795

Challenges in the care of individuals with severe primary insulin-like growth factor-I deficiency (SPIGFD): an international, multi-stakeholder perspective

Autor: Philippe F. Backeljauw, Mary Andrews, Peter Bang, Leo Dalle Molle, Cheri L. Deal, Jamie Harvey, Shirley Langham, Elżbieta Petriczko, Michel Polak, Helen L. Storr, Mehul T. Dattani

Publikováno v: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-12 (2023)

Abstract Background Severe primary insulin-like growth factor-I (IGF-I) deficiency (SPIGFD) is a rare growth disorder characterized by short stature (standard deviation score [SDS] ≤ 3.0), low circulating concentrations of IGF-I (SDS ≤ 3.0), and

Externí odkaz: https://doaj.org/article/fb8d3643879745efaa2cd385c0bcb5af

The Efficacy, Safety, and Pharmacology of a Ghrelin O-Acyltransferase Inhibitor for the Treatment of Prader-Willi Syndrome

Autor: Jennifer L Miller, André Lacroix, Lynne M Bird, Ashley H Shoemaker, Andrea Haqq, Cheri L Deal, Kristie A Clark, Michael H Ames, Jeffrey G Suico, Amparo de la Peña, Caroline Fortier

Publikováno v: J Clin Endocrinol Metab

Context Acylated ghrelin (AG) stimulates appetite and is elevated compared to its unacylated (UAG) counterpart in Prader-Willi syndrome (PWS). GLWL-01 is a selective, reversible inhibitor of ghrelin O-acyltransferase (GOAT), the enzyme that converts

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2e0ef9911a4392643726b04f8dae5cb7
https://doi.org/10.1210/clinem/dgac105

Efficacy and Safety of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Phase 3 Study

Autor: Cheri L Deal, Joel Steelman, Elpis Vlachopapadopoulou, Renata Stawerska, Lawrence A Silverman, Moshe Phillip, Ho-Seong Kim, CheolWoo Ko, Oleg Malievskiy, Jose F Cara, Carl L Roland, Carrie Turich Taylor, Srinivas Rao Valluri, Michael P Wajnrajch, Aleksandra Pastrak, Bradley S Miller

Publikováno v: The Journal of clinical endocrinology and metabolism. 107(7)

Context Somatrogon is a long-acting recombinant human growth hormone (rhGH) in development for once-weekly treatment of children with growth hormone deficiency (GHD). Objective We aimed to compare the efficacy and safety of once-weekly somatrogon wit

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1f66df9daab29abb1a313f0506af8d89
https://pubmed.ncbi.nlm.nih.gov/35405011

Contributors

Autor: Sonir R. Antonini, Zain Awamleh, Sioban Bacon, Corin Badiu, Kathryn Beardsall, Ingrid J. Block-Kurbisch, Kristien Boelaert, Isabelle Bourdeau, Marcello D. Bronstein, Helen Budge, Manuela Cerbone, Marlene Chakhtoura, Pranesh Chakraborty, Diana Maria Chitimus, Gertrude Costin, Mehul T. Dattani, Diva D. De Leon, Cheri L. Deal, Johnny Deladoëy, Lois E. Donovan, Ghada El-Hajj Fuleihan, Denice S. Feig, Analía V. Freire, Qinqin Gao, Audrey Garneau, Andrea Glezer, Veronica Gomez-Lobo, Romina P. Grinspon, Victor Han, Russell C. Hovey, Raquel Soares Jallad, Venkata S. Jonnakuti, Aspasia Karalis, Harshini Katugampola, Asma Khalil, Md. Wasim Khan, Ahmed Khattab, Christopher S. Kovacs, T’ng Chang Kwok, Anne-Marie Laberge, André Lacroix, David C.W. Lau, Sarah Elizabeth Lawrence, Brian T. Layden, Diana Le Duc, Na Li, Xiang Li, Alexis Light, Bailin Liu, Xiyuan Lu, Eugenie R. Lumbers, Anne Macdonald, Marcio Carlos Machado, Elizabeth A. McGee, Sam A. Mesiano, Geetha Mukerji, Cathy M. Murray, Mithra L. Narasimhan, Maria New, Amanda L. Ogilvy-Stuart, Shalini Ojha, Christine J. Orr, Anca Maria Panaitescu, Georgios E. Papadakis, Francesca Gabriela Paslaru, Jonathan Paul, Gheorghe Peltecu, Jason Phung, Nelly Pitteloud, Jerilynn C. Prior, Zoe E. Quandt, Bethany Radford, Fernando S. Ramalho, Rodolfo A. Rey, María Gabriela Ropelato, Adrian Eugen Rosca, Christopher W. Rowe, Jessica A. Ryniec, Anna Sadovnikova, Kirsten E. Salmeen, Mark E. Samuels, Sahar Sherf, Jien Shim, Roger Smith, Diana E. Stanescu, Brett Stark, Monica F. Stecchini, Matthieu St-Jean, Jerome F. Strauss, Miao Sun, Michael E. Symonds, Jiaqi Tang, Rosemary Townsend, Suzana Elena Voiculescu, Julia Elisabeth von Oettingen, Leanne M. Ward, Declan Wayne, Catherine Takacs Witkop, John J. Wysolmerski, Cheng Xu, Zhice Xu, Jennifer M. Yamamoto, Jessica S. Yang, Steven L. Young, Run Yu, Ana-Maria Zagrean, Leon Zagrean, Mengshu Zhang, Xiuwen Zhou

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::219ab5323127e1d5c6ff67fdb9159e8b
https://doi.org/10.1016/b978-0-12-814823-5.09993-1