AI Article Synopsis
- Traditional outcome measures in DMD trials exclude young boys due to reliance on cooperation, but alternative assessments like the Bayley III, HFMSE, and NSAA show promise.
- A study assessed 24 infants and boys with DMD, revealing significantly low scores in motor, cognitive, and language development compared to typical children, indicating developmental delays.
- Results suggest that outcome assessments for young boys with DMD are feasible, with the Bayley III emerging as the most effective measure in this multicenter study.
Article Abstract
Therapeutic trials in Duchenne Muscular Dystrophy (DMD) exclude young boys because traditional outcome measures rely on cooperation. The Bayley III Scales of Infant and Toddler Development (Bayley III) have been validated in developing children and those with developmental disorders but have not been studied in DMD. Expanded Hammersmith Functional Motor Scale (HFMSE) and North Star Ambulatory Assessment (NSAA) may also be useful in this young DMD population. Clinical evaluators from the MDA-DMD Clinical Research Network were trained in these assessment tools. Infants and boys with DMD (n = 24; 1.9 ± 0.7 years) were assessed. The mean Bayley III motor composite score was low (82.8 ± 8; p ≤ .0001) (normal = 100 ± 15). Mean gross motor and fine motor function scaled scores were low (both p ≤ .0001). The mean cognitive comprehensive (p=.0002), receptive language (p ≤ .0001), and expressive language (p = .0001) were also low compared to normal children. Age was negatively associated with Bayley III gross motor (r = -0.44; p = .02) but not with fine motor, cognitive, or language scores. HFMSE (n=23) showed a mean score of 31 ± 13. NSAA (n = 18 boys; 2.2 ± 0.4 years) showed a mean score of 12 ± 5. Outcome assessments of young boys with DMD are feasible and in this multicenter study were best demonstrated using the Bayley III.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3743677 | PMC |
| http://dx.doi.org/10.1016/j.nmd.2013.04.005 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Maternal Omega-6/Omega-3 Concentration Ratio During Pregnancy and Infant Neurodevelopment: The ECLIPSES Study.
Nutrients
January 2025
Nutrition and Mental Health (NUTRISAM) Research Group, Universitat Rovira i Virgili, 43201 Reus, Spain.
Background: The balance of omega-6/omega-3 (-6/-3) is crucial for proper brain function as they have opposite physiological roles.
Objectives: To analyze the association between maternal serum ratios of -6/-3 in the first and third trimesters of pregnancy and the neurodevelopment of their children in the early days after birth in the population of Northern Spain's Mediterranean region.
Methods: Longitudinal study in which 336 mother-child pairs participated.
Predicting Adverse Neurodevelopmental Outcomes in Premature Neonates with Intrauterine Growth Restriction Using a Three-Layered Neural Network.
Diagnostics (Basel)
January 2025
Clinical and Surgical Department, Faculty of Medicine and Pharmacy, 'Dunarea de Jos' University, 800216 Galati, Romania.
: There is a constant need to improve the prediction of adverse neurodevelopmental outcomes in growth-restricted neonates who were born prematurely. The aim of this retrospective study was to evaluate the predictive performance of a three-layered neural network for the prediction of adverse neurodevelopmental outcomes determined at two years of age by the Bayley Scales of Infant and Toddler Development, 3rd edition (Bayley-III) scale in prematurely born infants by affected by intrauterine growth restriction (IUGR). : This observational retrospective study included premature newborns with or without IUGR admitted to a tertiary neonatal intensive care unit from Romania, between January 2018 and December 2022.
View Article and Find Full Text PDFBalancing precision and affordability in assessing infant development in large-scale mortality trials: secondary analysis of a randomised controlled trial.
Arch Dis Child Fetal Neonatal Ed
January 2025
NHMRC Clinical Trials Centre, The University of Sydney, Camperdown, New South Wales, Australia.
Objective: Large-scale mortality trials require reliable secondary assessments of impairment. We compared the Ages and Stages Questionnaire (ASQ-3), a screening tool self-administered by parents, in classifying impairment using the 'gold standard' Bayley Scales of Infant Development (Bayley-III), a diagnostic tool administered by trained assessors.
Design: Analysis of 405 children around 2 years corrected age from the Australian Placental Transfusion Study, a trial conducted over 8 years.
Predictive model of neurodevelopmental outcome in neonatal hypoxic ischemic encephalopathy.
Early Hum Dev
January 2025
Department of Pediatrics, Division of Neonatology, The Hospital for Sick Children and the University of Toronto, Ontario, Canada. Electronic address:
Objectives: To build an early, prognostic model for adverse outcome in infants with hypoxic ischemic encephalopathy (HIE) receiving therapeutic hypothermia (TH) based on brain magnetic resonance images (MRI), electrophysiological tests and clinical assessments were performed during the first 5 days of life.
Methods: Retrospective study of 182 neonates with HIE and managed with TH. The predominant pattern of HIE brain injury on MRI performed following cooling was scored by neuroradiologists.
Parental and Medical Classification of Neurodevelopment in Children Born Preterm.
Pediatrics
January 2025
Department of Pediatrics, University of British Columbia, Vancouver, British Columbia, Canada.
Background And Objectives: The likelihood and severity of neurodevelopmental impairment (NDI) affects critical health care decisions. NDI definitions were developed without parental perspectives. We investigated the agreement between parental vs medical classification of NDI among children born preterm.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!