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BioWorld - Thursday, May 14, 2026

Neonatal/pediatrics

Home » Topics » Disease categories and therapies » Neonatal/pediatrics
  • Pediatric hydrocephalus neurology brain swell
    May 11, 2026

    ASO therapy prevents hydrocephalus in a monogenic syndrome model

    Researchers from McGill University and collaborating institutions aimed to investigate whether oligonucleotides are a viable drug class to prevent hydrocephalus.
  • Brain genetics
    May 4, 2026

    AAV9-delivered AntagoNATs have preclinical efficacy as one-time treatment for Dravet syndrome

    Dravet syndrome is a rare, severe, lifelong developmental and epileptic encephalopathy that begins in infancy and is marked by prolonged, often fever-triggered seizures that are difficult to control. It is usually caused by mutations in the SCN1A gene and is associated with developmental delay, cognitive and behavioral impairment, and reduced life expectancy.
  • Brain pineal gland
    March 11, 2026
    By Mar de Miguel

    Three pediatric brain cancer types share a pineal gland origin

    Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma, retinoblastoma and Group 3 medulloblastoma – have been linked to their shared origin during pineal gland development. Scientists at St. Jude Children’s Research Hospital have identified the molecular signatures that drive these tumors from pinealocyte progenitor cells that conserve a common differentiation program, providing a shared therapeutic target for these three cancer types.
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