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Research

Health care utilization and costs for children and adults with duchenne muscular dystrophy

Annual economic cost of Duchenne Muscular Dystrophy was found to be high, reflecting a significant socioeconomic burden, especially in boys who reach adulthood

Research

Heterogeneity in mechanisms of emergent resistance in pediatric T-cell acute lymphoblastic leukemia

Biological changes associated with T-ALL relapse and resistance are stochastic and highly individual

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Common variation contributes to the genetic architecture of social communication traits

Social communication difficulties represent an autistic trait that is highly heritable and persistent during the course of development.

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A "bottom-up" approach to aetiological research in autism spectrum disorders

Autism spectrum disorders (ASD) are currently diagnosed in the presence of impairments in social interaction and communication, and a restricted range of...

Research

The Differential Influence of Contextual Risks on Psychosocial Functioning and Participation of Australian Aboriginal Youth

Methodological issues and implications for interventions to support young Aboriginal people's adaptation are discussed.

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Novel oncogenic PDGFRA mutations in pediatric high-grade gliomas

The outcome for children with high-grade gliomas (HGG) remains dismal, with a 2-year survival rate of only 10% to 30%.

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Recommendations from a consensus development workshop on the diagnosis of fetal alcohol spectrum disorders in Australia.

Fetal alcohol spectrum disorders (FASD) are underdiagnosed in Australia, and health professionals have endorsed the need for national guidelines for diagnosis.

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Hypomethylation of the CTGF gene locus is a common feature of paediatric pre-B acute lymphoblastic leukaemia

We identified consistent hypomethylation of the CTGF locus in primary pre-B ALL specimens regardless of CTGF expression.

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Evaluating a community-based early childhood education and development program in Indonesia: study protocol for a pragmatic cluster randomized controlled trial

The aim of the trial is to evaluate a community-based early education and development program launched by the Government of Indonesia.

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Chest computed tomography: a validated surrogate endpoint of cystic fibrosis lung disease?

Clinical trials for the treatment of cystic fibrosis (CF) lung disease are important to test and optimise new therapeutic interventions.