Genetic conditions of short stature: A review of three classic examples

Autor: Merlin G. Butler, Bradley S. Miller, Alicia Romano, Judith Ross, M. Jennifer Abuzzahab, Philippe Backeljauw, Vaneeta Bamba, Amrit Bhangoo, Nelly Mauras, Mitchell Geffner

Publikováno v: Frontiers in Endocrinology, Vol 13 (2022)

Noonan, Turner, and Prader-Willi syndromes are classical genetic disorders that are marked by short stature. Each disorder has been recognized for several decades and is backed by extensive published literature describing its features, genetic origin

Externí odkaz: https://doaj.org/article/be5ba1ffb43a4445bee8749412bcebc7

Cambridge hybrid closed-loop algorithm in children and adolescents with type 1 diabetes: a multicentre 6-month randomised controlled trial

Autor: Julia Ware, MD, Charlotte K Boughton, PhD, Janet M Allen, RN, Malgorzata E Wilinska, PhD, Martin Tauschmann, PhD, Louise Denvir, MD, Ajay Thankamony, MPhil, Fiona M Campbell, MD, R Paul Wadwa, ProfMD, Bruce A Buckingham, ProfMD, Nikki Davis, MD, Linda A DiMeglio, ProfMD, Nelly Mauras, MD, Rachel E J Besser, PhD, Atrayee Ghatak, MD, Stuart A Weinzimer, ProfMD, Korey K Hood, ProfPhD, D Steven Fox, MD, Lauren Kanapka, MSc, Craig Kollman, PhD, Judy Sibayan, MPH, Roy W Beck, PhD, Roman Hovorka, ProfPhD, R Hovorka, C L Acerini, A Thankamony, J M Allen, C K Boughton, K Dovc, D B Dunger, J Ware, G Musolino, M Tauschmann, M E Wilinska, J F Hayes, S Hartnell, S Slegtenhorst, Y Ruan, M Haydock, J Mangat, L Denvir, SK Kanthagnany, J Law, T Randell, P Sachdev, M Saxton, A Coupe, S Stafford, A Ball, R Keeton, R Cresswell, L Crate, H Cripps, H Fazackerley, L Looby, H Navarra, C Saddington, V Smith, V Verhoeven, S Bratt, N Khan, L Moyes, K Sandhu, C West, R P Wadwa, G Alonso, G Forlenza, R Slover, L Towers, C Berget, A Coakley, E Escobar, E Jost, S Lange, L Messer, K Thivener, F M Campbell, J Yong, E Metcalfe, M Allen, S Ambler, S Waheed, J Exall, J Tulip, B A Buckingham, L Ekhlaspour, D Maahs, L Norlander, T Jacobson, M Twon, C Weir, B Leverenz, J Keller, N Davis, A Kumaran, N Trevelyan, H Dewar, G Price, G Crouch, R Ensom, L Haskell, LM Lueddeke, N Mauras, M Benson, K Bird, K Englert, J Permuy, K Ponthieux, J Marrero-Hernandez, L A DiMeglio, H Ismail, H Jolivette, J Sanchez, S Woerner, M Kirchner, M Mullen, M Tebbe, R EJ Besser, S Basu, R London, T Makaya, F Ryan, C Megson, J Bowen-Morris, J Haest, R Law, I Stamford, A Ghatak, M Deakin, K Phelan, K Thornborough, J Shakeshaft, S A Weinzimer, E Cengiz, J L Sherr, M Van Name, K Weyman, L Carria, A Steffen, M Zgorski, J Sibayan, R W Beck, S Borgman, J Davis, J Rusnak, A Hellman, P Cheng, L Kanapka, C Kollman, C McCarthy, S Chalasani, K K Hood, S Hanes, J Viana, M Lanning, D S Fox, G Arreaza-Rubin, T Eggerman, N Green, R Janicek, D Gabrielson, S H Belle, J Castle, J Green, L Legault, S M Willi, C Wysham

Publikováno v: The Lancet: Digital Health, Vol 4, Iss 4, Pp e245-e255 (2022)

Summary: Background: Closed-loop insulin delivery systems have the potential to address suboptimal glucose control in children and adolescents with type 1 diabetes. We compared safety and efficacy of the Cambridge hybrid closed-loop algorithm with us

Externí odkaz: https://doaj.org/article/9bf15c000c1f41e1bce13d4bb5d7e2c3

An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment

Autor: Zvi Zadik, Nataliya Zelinska, Violeta Iotova, Yulia Skorodok, Oleg Malievsky, Nelly Mauras, Srinivas Rao Valluri, Aleksandra Pastrak, Ron Rosenfeld

Publikováno v: Journal of Pediatric Endocrinology and Metabolism. 36:261-269

Objectives Somatrogon is a long-acting recombinant human growth hormone (GH) employed as a once-weekly treatment for children with GH deficiency (GHD). A 12-month, phase 2 study of once-weekly somatrogon vs. once-daily GH (Genotropin®) was initiated

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::b18bcfd7058927094226e11c5427766c
https://doi.org/10.1515/jpem-2022-0359

Positive Impact of the Bionic Pancreas on Diabetes Control in Youth 6–17 Years Old with Type 1 Diabetes: A Multicenter Randomized Trial

Autor: Laurel H, Messer, Bruce A, Buckingham, Fran, Cogen, Mark, Daniels, Greg, Forlenza, Rabab Z, Jafri, Nelly, Mauras, Andrew, Muir, R Paul, Wadwa, Perrin C, White, Steven J, Russell, Edward R, Damiano, Firas H, El-Khatib, Katrina J, Ruedy, Courtney A, Balliro, Zoey, Li, Martin Chase, Marak, Peter, Calhoun, Roy W, Beck

Publikováno v: Diabetes Technology & Therapeutics. 24:712-725

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::389b245d9407c818b26cc817b5006783
https://doi.org/10.1089/dia.2022.0201.pub

The Insulin-Only Bionic Pancreas Improves Glycemic Control in Non-Hispanic White and Minority Adults and Children with Type 1 Diabetes

Autor: the Bionic Pancreas Research Group, Nelly Mauras, Keisha Bird, Peter Calhoun, Ryan Bailey, Viral N. Shah, Roy W. Beck, Edward R. Damiano, Steven J. Russell, Luz E. Castellanos

Objective: We evaluated the performance of the iLet® bionic pancreas (BP) in non-Hispanic Whites (‘Whites’) and in Blacks, Hispanics, and others (‘Minorities’). Research Design and Methods: A multicenter, randomized controlled trial evaluate

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::01bd85b07d796f5526459648b7a2dc17
https://doi.org/10.2337/figshare.22221280