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Showing results for "Neuromuscular disorders "

Septo-optic dysplasia and gastroschisis: trends in birth prevalence and association with maternal age

This study aims to describe the risk factors and trends in birth prevalence of septo-optic dysplasia (SOD) and gastroschisis between 1980 and 2023. This descriptive, population-based study of SOD and gastroschisis used Western Australian Register of Developmental Anomalies data from 1980 to 2023. Birth prevalence was calculated using Midwives Notification System data for all births after 20 weeks gestation.

Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trial

CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes. 

How Well Does the EQ-5D-Y-5L Describe Children With Intellectual Disability?: “There's a Lot More to My Child Than That She Can't Wash or Dress Herself.”

The EQ-5D-5L is a generic health utility instrument for measuring health-related quality of life (HRQoL), with self-report and proxy report versions for children (EQ-5D-Y-5L). Children with intellectual disability (ID) are a heterogeneous population whose impairments and comorbidities place them at risk of poor HRQoL. This study aimed to describe the content validity and suitability for children with ID of a proxy report version of the EQ-5D-Y-5L as seen by their caregivers.

Caregiver-reported quality of life in individuals with developmental and epileptic encephalopathy and other severe neurodevelopmental encephalopathies

Information on factors contributing to quality of life (QOL) informs meaningful patient-centred care. We evaluated factors influencing QOL in individuals with developmental and epileptic encephalopathy (DEE) and other severe neurodevelopmental encephalopathy conditions using hypothesis-free regression tree analysis.

Sleep-disordered breathing in Australian children with Prader-Willi syndrome following initiation of growth hormone therapy

In children with Prader-Willi syndrome (PWS), growth hormone (GH) improves height and body composition; however, may be associated with worsening sleep-disordered breathing (SDB). Some studies have reported less SDB after GH initiation, but follow-up with polysomnography is still advised in most clinical guidelines.

Unpacking the complex nature of the autism epidemic

The etiology of autism spectrum disorders is unknown but there are claims of increasing prevalence in many countries.

Revision of the Australian guidelines to reduce health risks from drinking alcohol

The Australian guidelines to reduce health risks from drinking alcohol were released in 2020 by the National Health and Medical Research Council. Based on the latest evidence, the guidelines provide advice on how to keep the risk of harm from alcohol low.

Language and literacy in the context of early life adversity

This chapter considers the role of different forms of psychosocial adversity as risk factors for compromised language and literacy development in childhood and adolescence. Language skills play a central role in the development of prosocial interpersonal skills as well as promoting academic success through the transition to literacy.

“Society really does not like people with psychosis”: A thematic analysis of the stigma and self-stigma experiences of young people at-risk for psychosis

Stigma and self-stigma reduce self-esteem and increase hopelessness and suicidality. While psychotic disorders are widely recognized as the most stigmatizing of all mental health disorders, there is a dearth of research investigating how stigma and self-stigma are experienced by young people at ultra-high risk (UHR) for psychosis.

Autism Early Intervention Providers: Their Priorities, Use of Empirically Supported Practices, and Professional Development Needs

Autism early intervention research has indicated a research-to-practice gap, including continued use of practices with inadequate research support, and insufficient use of empirically supported practices. The present study explored the processes and mechanisms through which providers working with young children on the autism spectrum learn, select, and implement the various practices in their clinical repertoires.