Object: Infantile and late-infantile neuronal ceroid lipofuscinoses (NCLs) are invariably fatal lysosomal storage diseases associated with defects in lysosomal enzyme palmitoyl-protein thioesterase 1 (PPT-1) or tripeptidyl peptidase 1 (TPP1) activity. Previous preclinical studies have demonstrated that human CNS stem cells (HuCNS-SCs) produce both PPT-1 and TPP1 and result in donor cell engraftment and reduced accumulation of storage material in the brain when tested in an NCL mouse model.
Methods: HuCNS-SC transplantation was tested in an open-label dose-escalation Phase I clinical trial as a potential treatment for infantile and late-infantile NCL.
In the care of children with autism spectrum disorders (ASD), medical treatment is typically considered an adjunct to educational and behavioral interventions. Nonetheless, large proportions of children with ASD are managed medically and receive both pharmacologic and complementary-alternative medicine (CAM) treatments. Although many medical treatments have been studied in children with ASD, studies vary widely in terms of the sample, sample size, research design, purposes of treatment, and measurements of change.
View Article and Find Full Text PDFThe mechanism behind constraint-induced movement therapy (constraint therapy) success is unknown. Study objectives were to evaluate cortical change after modified constraint therapy and explore a novel approach to quantify developmental disregard. Five participants underwent modified constraint therapy.
View Article and Find Full Text PDFObjective: Because limited long-term outcome data exist for infants born at 32 to 36 weeks gestation, we compared school outcomes between 32- to 33-week moderate preterm (MP), 34-36 week late preterm (LP) and full-term (FT) infants.
Study Design: A total of 970 preterm infants and 13 671 FT control subjects were identified from the Early Childhood Longitudinal Study-Kindergarten Cohort. Test scores, teacher evaluations, and special education enrollment from kindergarten (K) to grade 5 were compared.
Constraint-induced movement therapy improves motor function in the affected hand of children with hemiplegic cerebral palsy and results in cortical changes in adults with stroke. This study measured clinical improvement and cortical reorganization in a child with hemiplegia who underwent modified constraint-induced movement therapy for 3 weeks. Clinical, functional magnetic resonance imaging and magnetoencephalography measurements were done at baseline, after therapy, and 6 months after therapy.
View Article and Find Full Text PDFObjective: To measure the association between daytime bottle-feeding and iron depletion in young children.
Design: Cross-sectional design with concurrent measurement of exposure and outcome. The exposure was the current container (bottle or cup) used for daytime milk consumption.