Articles: ninos.
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New gene therapy approaches have emerged as promising treatment options for rare congenital disorders and certain tumor entities for which previously only procedures of limited curative potential had been available, if at all. ⋯ The first gene treatments have now been approved and bring hope of long-term therapeutic benefit after a single administration. The numbers of patients who come into question for specific therapies are often low, so that many different aspects- generation of evidence on efficacy and safety, determining indications, performance of the treatment, pricing-bring new challenges.
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Current evidence indicates that children are relatively spared from direct COVID-19-related morbidity and mortality, but that the indirect effects of the pandemic pose significant risks to their health and wellbeing. ⋯ Multiple indicators demonstrated disruption in service access, service delivery and child wellbeing. Further studies are needed to establish the intermediate- and long-term impacts of the COVID-19 outbreak on child health, as well as strategies to mitigate these.