Neuropsychological Late Effects and Quality-of-Life Outcomes in Pediatric Brain Tumor Survivors: Role of Pediatric Neurologists in Monitoring and Management.

Pediatric brain tumor (PBT) survivors are at significantly increased risk of cognitive, psychosocial, and educational/vocational sequelae that impact health-related quality of life. These complications and health morbidities result in high burden on survivors and their families, particularly those already vulnerable to disparities in health care access and outcomes. Since neurological comorbidities are common in this population, neurologists are uniquely positioned to screen, treat identified symptoms, and connect families with services and resources.

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder characterized by deficient synthesis of dopamine and serotonin. It presents in early infancy, and causes severe developmental disability and lifelong motor, behavioral, and autonomic symptoms including oculogyric crises (OGC), sleep disorder, and mood disturbance. We investigated the safety and efficacy of delivery of a viral vector expressing AADC (AAV2-hAADC) to the midbrain in children with AADC deficiency (ClinicalTrials.

Cognitive Functioning and Academic Performance in Elementary School Children with Anxious/Depressed and Withdrawn Symptoms.

RATIONALE: Few studies have evaluated the relationship between depressive symptomatology and neuropsychological performance in children without symptomatic depression. OBJECTIVES: This study determined the relationship between anxious/depressed and withdrawn symptoms and performance on cognitive and academic achievement measures. METHODS: 335 Caucasian and Hispanic children aged 6 to 11 years who participated in the Tucson Children's Assessment of Sleep Apnea (TuCASA) study were administered a comprehensive neuropsychological battery measuring cognitive functioning and academic achievement.