ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica

Page 1 of about 6 results

ey0019.13-5 | Improving access to healthcare in pediatric endocrinology and diabetes | ESPEYB19

13.5. Access to insulin products in Pakistan: a national scale cross-sectional survey on prices, availability, and affordability

A Saeed , K Lambojon , H Saeed , Z Saleem , N Anwer , M Majid Aziz , W Ji , W Liu , C Chen , C Yang , Y Fang , Z-U-D Babar

yufang@mail.xjtu.edu.cn Front Pharmacol 2022 13:820621. doi: 10.3389/fphar.2022.820621Brief Summary: This national cross-sectional survey in Pakistan found poor availability of insulin in the public sector, even for brands manufactured by domestic companies.Access to insulin remains a major issue worldwide in low- and middl...
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ey0019.5-8 | Advances in clinical practice | ESPEYB19

5.8. Growth in achondroplasia including stature, weight, weight-for-height and head circumference from CLARITY: achondroplasia natural history study-a multi-center retrospective cohort study of achondroplasia in the US

JE Hoover-Fong , KJ Schulze , AY Alade , MB Bober , E Gough , SS Hashmi , JT Hecht , JM Legare , ME Little , P Modaff , RM Pauli , DF Rodriguez-Buritica , ME Serna , C Smid , C Liu , J McGready

Orphanet J Rare Dis. 2021 Dec 23;16(1):522.Abstract: https://pubmed-ncbi-nlm-nih-gov/34949201/In Brief: Based on over 37,000 length/height, weight and head circumference measures from 1374 patients with achondroplasia in the USA, updated length/height-for-age, weight-for-age, head circumference-for-age (HC) and weight-for-height curves were generated for children up to 18 years of age (HC u...
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ey0019.7-5 | Clinical Guidance | ESPEYB19

7.5. Analysis of early-life growth and age at pubertal onset in US children

IM Aris , W Perng , D Dabelea , JM Ganiban , C Liu , K Marceau , OC Robertson , CW Hockett , NL Mihalopoulos , X Kong , MM Herting , TM O'Shea , ET Jensen , MF Hivert , E Oken

Program Collaborators for Environmental Influences on Child Health OutcomesJAMA Netw Open. 2022 Feb 1;5(2):e2146873. doi: 10.1001/jamanetworkopen.2021.46873. PMID: 35119461https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2788643/Brief Summary: This study describes the correlation between...
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ey0019.12-11 | Hyperlipidemia | ESPEYB19

12.11. Identification and evaluation of a lipid-lowering small compound in preclinical models and in a Phase I trial

H. Wang J, Zhao J, Yan C, Xi C, Wu C, Zhao J, Li F, Ding Y, Zhang R, Qi S, Li X, Liu C, Hou W, Chen H, Wang Y, Wu D, Chen K, Jiang H, Huang H, Liu

Cell Metabolism 2022;34(5):667-80.e6. doi: 10.1016/j.cmet.2022.03.006Brief Summary: In preclinical models and a phase 1 trial, a powerful new lipid small molecule was shown to act through a mechanism distinct from those of known hypolipidemic agents. Targeting HNF-1α may be a new therapeutic strategy.Comment: Familial hypercholesterolemia (FH) is the most common...
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ey0019.10-15 | Pathogenesis | ESPEYB19

10.15. Single-cell multi-omics analysis of human pancreatic islets reveals novel cellular states in type 1 diabetes

M Fasolino , GW Schwartz , AR Patil , A Mongia , ML Golson , YJ Wang , A Morgan , C Liu , J Schug , J Liu , M Wu , D Traum , A Kondo , CL May , N Goldman , W Wang , M Feldman , JH Moore , AS Japp , MR Betts , Consortium HPAP , RB Faryabi , A Naji , KH Kaestner , G Vahedi

Nat Metab. 2022 Feb;4(2):284-299. https://pubmed.ncbi.nlm.nih.gov/35228745/Brief Summary: This study used three high-throughput single-cell technologies to generate a pancreatic islet cell atlas from 24 organ donors with type 1 diabetes (T1D), autoantibody positive and healthy donors. The most remarkable finding was that a subset of exocrine ductal cells appears to acquire a signature of to...
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ey0020.4-3 | Sexuality, Fertility and Fertility Optimization in DSD | ESPEYB20

4.3. AAV-mediated gene therapy produces fertile offspring in the Lhcgr-deficient mouse model of Leydig cell failure

K Xia , F Wang , X Lai , L Dong , P Luo , S Zhang , C Yang , H Chen , Y Ma , W Huang , W Ou , Y Li , X Feng , B Yang , C Liu , Z Lei , X Tu , Q Ke , FF Mao , C Deng , AP Xiang

Brief summary: In this in vivo study, Xia et al. demonstrate that AAV-mediated gene therapy recovers testosterone levels, restarts sexual development, restores spermatogenesis, and produces fertile offspring in a mouse model of Leydig cell failure (LCF).A null mutation in the gene encoding luteinizing hormone/choriogonadotrophin receptor (Lhcgr) causes a hereditary LCF in mice which is characterized by a reduction in testosterone levels...
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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