paediatrics

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Millions of children around the world are at risk of serious infectious diseases because immunisation programmes have stalled, says a new study.

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Biogen has started a new phase 3 trial that aims to extend the use of its Friedreich's ataxia drug Skyclarys to patients as young as two.

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Novo Nordisk's long-acting injectable Sogroya has been backed for NHS use in young people with growth hormone deficiency.

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In a medical breakthrough, a child diagnosed with a rare genetic disorder has received a bespoke CRISPR gene editing therapy to correct the mutation.

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Should treatment of neurodegenerative disorder SMA with Roche's Evrysdi start even before birth? One case study suggests so.

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Sensorion's gene therapy for otoferlin-related hearing loss has shown early signs of improving hearing in two toddlers in a phase 1/2 trial.