The purpose of this study was to test the feasibility and short-term outcomes of Asthma: It's a Family Affair!, a school-based intervention for adolescents with asthma and their caregivers. Twenty-four ethnic minority families with a middle school student with asthma were randomized to immediate intervention or no-treatment control. Intervention students received six group sessions on prevention and management of asthma. Caregivers received five group sessions teaching child-rearing skills to support the youth's autonomy and asthma self-management. All students attended all sessions; caregivers attended an average of three. Two months post-intervention, relative to controls, intervention caregivers reported better problem-solving with children. Intervention students were more responsible for carrying medication, took more prevention steps, and woke fewer nights from asthma. The intervention resulted in positive short-term changes in family relations, asthma management by students, and health status.
BackgroundMucopolysaccharidosis type III (MPS III, Sanfilippo syndrome) is a lysosomal storage disorder caused by a deficiency of one of the enzymes involved in the degradation of heparan sulfate.MPS III is characterized by progressive mental deterioration resulting in severe dementia. A number of potentially disease-modifying therapies are studied. As preservation of cognitive function is the ultimate goal of treatment, assessment of cognitive development will be essential in order to evaluate treatment efficacy. However, no large scale studies on cognitive levels in MPS III patients, using formal psychometric tests, have been reported.MethodsWe aimed to assess cognitive development in all 73 living patients with MPS III in the Netherlands.ResultsCognitive development could be assessed in 69 patients. In 39 of them developmental level was estimated > 3 months and formal psychometric testing was attempted. A remarkable variation in the intellectual disability was detected.ConclusionsDespite special challenges encountered, testing failed in only three patients. The observed broad variation in intellectual disability, should be taken into account when designing therapeutic trials.
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