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Child Health nieuws

Child Health nieuws

New gene therapy reimbursed for children with MLD

New gene therapy is reimbursed for children with a very rare metabolic disease (MLD). MLD is a severe genetic disease caused by a mistake in DNA. This gene therapy is the only effective treatment for a severe variant of MLD in which children deteriorate rapidly. The Dutch government recently started reimbursing this therapy for patients who have no symptoms yet.

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Symposium: If moving is a concern

Maaike Sprong

Young children with severe congenital heart defects have an increased risk of developing brain damage due to delayed brain development. As a result, long-term developmental problems frequently occur.

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Precision medicine to treat effectively

Marcel Kool

Marcel Kool, professor of Childhood brain tumors at UMC Utrecht, delivered his oration on Wednesday 24 January, titled "Pediatric Brain Tumors - WHO cares!?" on the different types of brain tumors in children, developments in precision medicine and the search for effective treatments.

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Substantial grant awarded for smart alarm in ICU

UMC Utrecht is coordinating the Smart Alarm Management Study. This investigates ways in which beeping medical devices around the patient's bedside can be used differently. Using smarter algorithms, the devices can transform into a silent alarm chain that informs the caregiver in a proper and timely manner. Eight tons have been allocated for this study, which is being conducted as part of Smart And Silent ICU (SASICU). This is a €17 million public-private funded research project. Several other European teaching hospitals and Innovation Partners Dräger and Ascom are involved.

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AI forecasts outcomes in very premature babies

Researchers at UMC Utrecht have developed an AI model to predict long-term outcome in extremely premature babies early in life. The model can identify which infants might face intellectual disability as they grow. When further developed, it could offer crucial insights for healthcare providers as well as valuable information for parents about their child’s expected developmental journey. The results of the study have been published in The Lancet Digital Health.

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Newly described mechanism offers opportunities for treatment of cystic fibrosis

People with cystic fibrosis have a compromised defense against pathogens in the lungs, due to the accumulation of thick mucus in their lungs in which pathogens thrive. Apart from the mucus, the fluids lining the airway surfaces are also too acidic and that inhibits additional antimicrobial defense mechanisms against pathogens. Researchers from Newcastle University and University Medical Center Utrecht have described a new cellular mechanism that influences this acidity using miniature airways, and discovered two already approved drugs that could potentially be used to change the acidity in people with CF.

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Pay attention to late effects of childhood cancer

Leontien Kremer

Leontien Kremer, professor of late effects after childhood cancer at Utrecht University delivered her oration titled 'LIFE: now and LATER' on caring for survivors of childhood cancer with late effects, health problems after treatment.

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Utrecht collaboration strengthens care and research

Children with a tumour near the hypothalamus - the hormone centre in the brain - may suffer from hormone imbalances. This can be caused by the tumour, but symptoms can also worsen after treatment through surgery or radiotherapy. If the hypothalamus is damaged, in addition to a hormone imbalance, there is also a disturbed sense of hunger and thirst, heat regulation and day-night rhythms. As a result, they may suffer from severe obesity and chronic fatigue after treatment.

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Investigating genetic kidney failure in young people

Team erfelijke nierziekten

Albertien van Eerde, clinical geneticist and associate professor translational nephrogenetics, Gisela Slaats, assistant professor experimental nephrology, and Marc Lilien, paediatric nephrologist at UMC Utrecht, played a crucial role in securing Utrecht's participation in TheRaCil consortium. This will take research into kidney failure in young people to a higher level. The researchers involved from 15 institutes from six countries are working closely together as they integrate their expertise and the various large European ciliopathy biobanks and databases.

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'Paediatric radiologist: we contribute to optimal care'

Portrait of Rutger Jan Nievelstein, professor of Paediatric Radiology at UMC Utrecht.

Well-supported guidelines for paediatric radiological imaging are needed to ensure the best care for children, Professor of Paediatric Radiology Rutger Jan Nievelstein recently stated in his inaugural address. This requires sound scientific research. After all, children have a different range of conditions than adults, such as congenital defects, hereditary and metabolic diseases, and certain types of tumours. The new chair that will strengthen this scientific research falls under the strategic theme Child Health and supports the enhancers image-guided interventions and integral complex care for children.

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