FDA approves Voxzogo for children, first drug to improve growth in most common form of dwarfism

2021 ◽  
Vol 24 (1) ◽  
pp. 8-8
Keyword(s):  
2015 ◽  
Author(s):  
Werner Zenz ◽  
Andreas Trobisch ◽  
Daniela Klobassa ◽  
Alexander Binder ◽  
Matthias Sperl ◽  
...  

2020 ◽  
Author(s):  
Laurie John Hannigan ◽  
Ragna Bugge Askeland ◽  
Helga Ask ◽  
Martin Tesli ◽  
Elizabeth Corfield ◽  
...  

BackgroundEarly developmental milestones, such as the age at first walking or talking, are associated with later diagnoses of neurodevelopmental disorders, but the relationship to genetic risk for neurodevelopmental disorders are unknown. Here, we investigate associations between genetic liability to autism spectrum disorder (autism), attention deficit hyperactivity disorder (ADHD), and schizophrenia and attainment of early-life language and motor development milestones.MethodsWe use data from a genotyped sub-set (N = 15 205) of children in the Norwegian Mother, Father and Child Cohort Study (MoBa). In this sample, we calculate polygenic scores for autism; ADHD and schizophrenia and predict maternal reports of children’s age at first walking and talking, motor delays at 18 months, language delays at 3 years, and a generalized measure of concerns about development. We use linear and probit regression models in a multi-group framework to test for sex differences.ResultsADHD polygenic scores predicted earlier walking age in both males and females (β=-0.037, pFDR=0.001), and earlier first use of sentences (β=-0.087, pFDR=0.032) but delayed language development at 3 years in females only (β=0.194, pFDR=0.001). Additionally, we found evidence that autism polygenic scores were associated with later walking (β=0.027, pFDR=0.024) and motor delays at 18 months (β = 0.065, pFDR=0.028). Schizophrenia polygenic scores were associated with a measure of general concerns about development at 3 years in females only (β=0.132, pFDR=0.024).ConclusionsGenetic liabilities for neurodevelopmental disorders show some specific associations with measures of early motor and language development in the general population, including the age at which children first walk and talk. Associations are generally small and occasionally in unexpected directions. Sex differences are evident in some instances, but clear patterns across different polygenic scores and outcomes are hard to discern. These findings suggest that genetic susceptibility for neurodevelopmental disorders is manifested in the timing of developmental milestones in infancy.


2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Ying Bi ◽  
Yifan Zhu ◽  
Xiao Ma ◽  
Jiejing Xu ◽  
Yun Guo ◽  
...  

AbstractNow there is no clinical scale for early prediction of refractory Mycoplasma pneumoniae pneumonia (RMPP). The aim of this study is to identify indicators and develop an early predictive scale for RMPP in hospitalized children. First we conducted a retrospective cohort study of children with M. pneumoniae pneumonia admitted to Children’s Hospital of Nanjing Medical University, China in 2016. Children were divided into two groups, according to whether their pneumonia were refractory and the results were used to develop an early predictive scale. Second we conducted a prospective study to validate the predictive scale for RMPP in children in 2018. 618 children were included in the retrospective study, of which 73 with RMPP. Six prognostic indicators were identified and included in the prognostic assessment scale. The sensitivity of the prognostic assessment scale was 74.0% (54/73), and the specificity was 88.3% (481/545) in the retrospective study. 944 children were included in the prospective cohort, including 92 with RMPP, the sensitivity of the prognostic assessment scale was 78.3% (72/92) and the specificity was 86.2% (734/852). The prognostic assessment scale for RMPP has high diagnostic accuracy and is suitable for use in standard clinical practice.


2014 ◽  
Vol 201 (5) ◽  
pp. 243-243
Author(s):  
Tania Janusic

1975 ◽  
Vol 2 (2) ◽  
pp. 251-260 ◽  
Author(s):  
Svenka Savić

This note attempts to clarify the early acquisition of the interrogative system, with data from Serbo-Croatian. The subject is approached from an angle that has hitherto not received sufficient treatment: adult-child interaction in direct communication. The process of question acquisition was observed in a first-born pair of dizygotic twins – a girl, Jasmina, and a boy, Danko – between I; I and 3; 0, the observation beginning a month prior to the time when the children first began to produce questions. The material was transcribed during weekly two-hour sessions in the home of the subjects.


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