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Paediatrics
5 articles · Newest source date first
Clinical trialChildhood tooth decay: trial supports a topical approach to stopping progression
NIH highlighted evidence on silver diamine fluoride for cavities in young children.
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FDA expanded gene-therapy approvalCRISPR-based Casgevy expands to children from age two with sickle cell disease or transfusion-dependent β-thalassaemia
The age expansion brings gene-edited autologous stem-cell therapy to much younger children with severe inherited blood disorders.
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FDA accelerated approvalTzield becomes the first FDA-approved treatment aimed at preserving insulin production after recent stage 3 type 1 diabetes diagnosis in children
The anti-CD3 therapy now has a new indication intended to slow loss of endogenous insulin production in recently diagnosed pediatric type 1 diabetes.
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First gene therapy approved for OTOF-associated inherited hearing loss
Otarmeni uses a dual-AAV system to deliver functional OTOF genetic material directly to inner-ear hair cells.
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First approved therapy aimed at neurological manifestations of Hunter syndrome
Avlayah is designed to address a major limitation of conventional enzyme therapy: reaching the central nervous system.
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