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Outcomes of latent rheumatic heart disease: External validation of a simplified score in patients with and without secondary prophylaxis

Secondary antibiotic prophylaxis reduces progression of latent rheumatic heart disease (RHD) but not all children benefit. Improved risk stratificati…

Psychosocial wellbeing, parental concerns, and familial impact of children with developmental coordination disorder

Developmental Coordination Disorder (DCD) is a neurodevelopmental condition impacting motor skill acquisition and competence. While previous studies…

Performance Accuracy of Wrist-Worn Oximetry and Its Automated Output Parameters for Screening Obstructive Sleep Apnea in Children

Obstructive sleep apnea (OSA) increases the risk of perioperative adverse events in children. While polysomnography remains the reference standard fo…

“Food faddists and pseudoscientists!”: Reflections on the history of resistance to ultra-processed foods

The term 'ultra-processed food' emerged in the 1980s, mostly used in reference to highly-processed convenience foods and snacks, often energy-dense,…

Presence of onco-fetal neighborhoods in hepatocellular carcinoma is associated with relapse and response to immunotherapy

Onco-fetal reprogramming of the tumor ecosystem induces fetal developmental signatures in the tumor microenvironment, leading to immunosuppressive fe…

Normative Modeling of Brain Morphometry in Clinical High Risk for Psychosis

The lack of robust neuroanatomical markers of psychosis risk has been traditionally attributed to heterogeneity. A complementary hypothesis is that v…

Egg-sensitised infants have elevated CD4+ effector memory T regulatory cells from birth

IgE-mediated sensitisation to egg is common in infants. In some cases, the processes leading to egg sensitisation are established in early life, even…

CRISPR-Cas9-generated PTCHD1 2489T>G stem cells recapitulate patient phenotype when undergoing neural induction

An estimated 3.5%-5.9% of the global population live with rare diseases, and approximately 80% of these diseases have a genetic cause. Rare genetic d…

Joint developmental trajectories of internalizing and externalizing problems from mid-childhood to late adolescence and childhood risk factors: Findings from a prospective pre-birth cohort

There is limited evidence on heterogenous co-developmental trajectories of internalizing and externalizing problems from childhood to adolescence and…

FDA Patient-Focused Drug Development Guidances: Considerations for Trial Readiness in Rare Developmental and Epileptic Encephalopathies

Developmental and epileptic encephalopathies (DEE) are rare, often monogenic neurodevelopmental conditions. Most affected individuals have refractory…