ISSN 1662-4009 (online)

ey0020.10-4 | New Drugs for Children with T2DM | ESPEYB20

10.4. Once-weekly dulaglutide for the treatment of youths with type 2 diabetes

SA Arslanian , T Hannon , P Zeitler , LC Chao , C Boucher-Berry , M Barrientos-Perez , E Bismuth , S Dib , JI Cho , D Cox , AWARD-PEDS Investigators Cox

Brief summary: This randomized, double-blind, phase 3 trial of youths with inadequately controlled T2D, dulaglutide treatment was superior to placebo in reducing the glycated hemoglobin level at 26 weeks.Comment: T2D in children and adolescents is an aggressive disease with early onset of complications, leading to significant morbidity and mortality. After three decades with no efficient treatment, there is finally light at the end of the tunnel.<p c...

ey0020.11-8 | Diabetes | ESPEYB20

11.8. Insulin thermostability in a real-world setting

S Pendsey , S James , TJ Garrett , AB Nord , S Pendsey , D Malmodin , G Karlsson , J Maniam , MA Atkinson , G Forsander , GD Ogle

Brief summary: The potency of insulin decreases with increasing temperatures. This pilot study compared the concentration and structure of commonly used types of insulin when exposed to high temperatures for prolonged periods of time.Manufacturers are required to provide information on how insulin vials and cartridges should be kept by the patient. These recommendations assume that a working fridge is available and that the medicine can be protected from...

ey0020.11-15 | Endocrinology | ESPEYB20

11.15. Genotype, mortality, morbidity, and outcomes of 3[beta]-hydroxysteroid dehydrogenase deficiency in Algeria

A Ladjouze , M Donaldson , I Plotton , N Djenane , K Mohammedi , V Tardy-Guidollet , D Mallet , K Boulesnane , Z Bouzerar , Y Morel , F Roucher-Boulez

Brief summary: This study describes the genetic and clinical characteristics of 3βHSD2 deficiency in children seen at a single center in Algeria. It describes clinical outcomes, including the frequency of adrenal rest tumors in this population.3β-hydroxysteroid dehydrogenase 2 deficiency (3βHSD2) is a rare form of congenital adrenal hyperplasia. This mixed longitudinal and cross-sectional study was performed in a single Algerian center bet...

ey0018.4-6 | Growth Hormone Therapy: Safety | ESPEYB18

4.6. Long-term mortality after childhood growth hormone treatment: the SAGhE cohort study

L Savendahl , R Cooke , A Tidblad , D Beckers , G Butler , S Cianfarani , P Clayton , J Coste , ACS Hokken-Koelega , W Kiess , CE Kuehni , K Albertsson-Wikland , A Deodati , E Ecosse , R Gausche , C Giacomozzi , D Konrad , F Landier , R Pfaeffle , G Sommer , M Thomas , S Tollerfield , GRJ Zandwijken , JC Carel , AJ Swerdlow

Lancet Diabetes Endocrinol. 2020;8(8):683–692. doi: 10.1016/S2213-8587(20)30163-7. PMID: 32707116SAGhE is a large independent European consortium including eight different countries (Belgium, France, Germany, Italy, The Netherlands, Sweden, Switzerland, and the UK) which was set up to evaluate the long-term safety of rhGH in a large cohort (>24 000) of young adult patients t...

ey0018.7-1 | Clinical Guidance | ESPEYB18

7.1. Cranial MRI abnormalities and long-term follow-up of the lesions in 770 girls with central precocious puberty

D Helvacıoğlu , S Demircioğlu Turan , T Guran , Z Atay , A Dağcınar , D Bezen , E Karakılıc Ozturan , F Darendeliler , A Yuksel , F Dursun , S Kılınc , S Semiz , S Abalı , M Yıldız , A Onder , A Bereket

J Clin Endocrinol Metab. 2021 Mar 25:dgab190. doi: 10.1210/clinem/dgab190. PMID: 33765130. https://academic.oup.com/jcem/article-abstract/106/7/e2557/6188450?redirectedFrom=fulltextIn brief: This multicentre cohort study explores the frequency, long-term outcomes and potential p...

ey0018.7-10 | Basic Science | ESPEYB18

7.10. Central ceramide signaling mediates obesity-induced precocious Puberty

V Heras , JM Castellano , D Fernandois , I Velasco , E Rodriguez-Vazquez , J Roa , MJ Vazquez , F Ruiz-Pino , M Rubio , R Pineda , E Torres , MS Avendano , A Paredes , L Pinilla , D Belsham , C Dieguez , F Gaytan , N Casals , M Lopez , M Tena-Sempere

Cell Metab. 2020 Dec 1;32(6):951–966.e8. 10.1016/j.cmet.2020.10.001. PMID: 33080217. https://www.sciencedirect.com/science/article/pii/S1550413120305349?via%3DihubIn brief: This study reveals the critical role of hypothalamic ceramide synthesis in the induction of precocious puberty in obe...

ey0019.4-11 | New Paradigms | ESPEYB19

4.11. Fine-tuning cardiac insulin-like growth factor 1 receptor signaling to promote health and longevity

M Abdellatif , V Trummer-Herbst , AM Heberle , A Humnig , T Pendl , S Durand , G Cerrato , SJ Hofer , M Islam , J Voglhuber , Pittol JM Ramos , O Kepp , G Hoefler , A Schmidt , PP Rainer , D Scherr , Lewinski D Von , E Bisping , JR McMullen , A Diwan , T Eisenberg , F Madeo , K Thedieck , G Kroemer , S Sedej

Circulation, 2022: Jun 21;145(25):1853-1866 PMID: 35616058Brief Summary: This translational study evaluated cardiac health and lifespan in two cardiomyocyte-specific transgenic mice with either enhanced or reduced IGF-1 signaling and in human cardiac biopsies from failing and nonfailing hearts. Increased IGF1R expression was related to better cardiac performance in young mice but faster decline of cardiac function with aging. Conversely reduced ...

ey0019.5-3 | Novel treatments for rare skeletal disorders | ESPEYB19

5.3. Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study

R Savarirayan , L Tofts , M Irving , WR Wilcox , CA Bacino , J Hoover-Fong , RU Font , P Harmatz , F Rutsch , MB Bober , LE Polgreen , I Ginebreda , K Mohnike , J Charrow , D Hoernschemeyer , K Ozono , Y Alanay , P Arundel , Y Kotani , N Yasui , KK White , HM Saal , A Leiva-Gea , F Luna-Gonzalez , H Mochizuki , D Basel , DM Porco , K Jayaram , E Fisheleva , A Huntsman-Labed , JRS Day

Genet Med. 2021 Dec;23(12):2443-2447.Abstract: https://pubmed-ncbi-nlm-nih-gov.proxy.kib.ki.se/34341520/In brief: In achondroplasia, longitudinal bone growth is inhibited resulting in severe, disproportionate short stature. In this open-label extension study of participants from the phase 3 study, daily subcutaneous injection of vosoritide during 104 weeks resulted in increa...

ey0019.6-5 | Basic and Genetic Research of DSD | ESPEYB19

6.5. Broad-spectrum XX and XY gonadal dysgenesis in patients with a homozygous L193S variant in PPP2R3C

D Cicek , N Warr , G Yesil , Eker H Kocak , F Bas , S Poyrazoglu , F Darendeliler , G Direk , N Hatipoglu , M Eltan , Abali Z Yavas , Tosun B Gurpinar , SB Kaygusuz , Menevse T Seven , D Helvacioglu , S Turan , A Bereket , R Reeves , M Simon , M Mackenzie , L Teboul , A Greenfield , T Guran

Eur J Endocrinol. 2021 Dec 1;186(1):65-72. PMID: 34714774, doi: 10.1530/EJE-21-0910.Brief Summary: This brief report describes a novel gene, PPP2R3C, in the pathogenesis of complete and partial XY and XX gonadal dysgenesis (GD).GD is a very rare condition with an estimated prevalence of 1–9 cases per 100,000 live-births. GD can be classified as either complete...

ey0019.7-8 | Basic Science | ESPEYB19

7.8. GnRH neurons recruit astrocytes in infancy to facilitate network integration and sexual maturation

G Pellegrino , M Martin , C Allet , T Lhomme , S Geller , D Franssen , V Mansuy , M Manfredi-Lozano , A Coutteau-Robles , V Delli , S Rasika , D Mazur , A Loyens , M Tena-Sempere , J Siepmann , FP Pralong , P Ciofi , G Corfas , AS Parent , SR Ojeda , A Sharif , V Prevot

Nat Neurosci. 2021 Dec;24(12):1660-1672. doi: 10.1038/s41593-021-00960-z. Epub 2021 Nov 18. PMID: 34795451. https://www.nature.com/articles/s41593-021-00960-zBrief Summary: This neuroanatomical and physiological study in mice demonstrates that GnRH neurons attract glial cells in their vicinity via cell–cell communication...