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NIHR Great Ormond Street Hospital Biomedical Research Centre

h-index: 39
Total citations: 6,565
Total papers: 337

Homepage: https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/

Recent papers from NIHR Great Ormond Street Hospital Biomedical Research Centre

The boundaries of best interests: The case of Townsend v EpsomClinical Ethics · 2026
Cell-resolved pharmacodynamic profiling reveals tissue- and cell-population-specific activity of antisense oligonucleotide chemical conjugates in vivo2026
Global Epidemiology of Neonatal and Infantile-Onset Epilepsies and Epilepsy Syndromes (GENIE)JAMA Neurology · 2026
Three-Year Follow-Up of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Long-Term Phase 3 EMBARK Trial ResultsNeurology and Therapy · 2026
Early and long-term patency of inferior vena cava patch reconstruction in children with renal tumour thrombusEJC Paediatric Oncology · 2026
A phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of vosoritide in children with hypochondroplasia: CANOPY HCH-3 study designTherapeutic Advances in Endocrinology and Metabolism · 2026
Local retraining mitigates domain shift in sepsis prediction: Lessons from translating a neonatal model to mixed intensive care data2026
Systematic assessment of the medical utility of radiology and diagnostic Artificial Intelligence in fracture detection (SAMURAI-fracture): a protocol for a multicentre cluster-randomised controlled trialBMJ Open · 2026
Gene therapy approaches for inborn errors of immunity: from bench to bedsideExpert Opinion on Biological Therapy · 2026
In-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editingMolecular Therapy Advances · 2026
Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudyMolecular Genetics and Metabolism Reports · 2026
Regional Variations in Mortality, Surgical Treatment, and Hospitalization in Children With Congenital Diaphragmatic Hernia: A European Population‐Based Data‐Linkage Cohort StudyBirth Defects Research · 2026
Educational attainment among primary school children with neurodisability: a population-based cohort study using linked education and health data from EnglandArchives of Disease in Childhood · 2026
Hospital admissions among adolescents with local authority care experience or special educational needs in England: a population-based cohort study using linked administrative data from health, education and social care servicesBMJ Public Health · 2026
Recommendations for the diagnosis and management of cln3 disease (batten disease) using the Delphi consensus methodologyOrphanet Journal of Rare Diseases · 2026
Diagnostic Yield of Post-Mortem Fetal Micro-CT for Central Nervous System Abnormalities.2026
Muscle transcriptome profiling reveals novel molecular pathways and biomarkers in laminin-α2 deficient patientsActa Neuropathologica Communications · 2026
Educational Attainment of Children With Major Congenital Anomalies During Primary School in England: A Population Cohort Study.2026
Open Fetal Versus Postnatal Repair of Spina Bifida Aperta—A Comparison of Neonatal OutcomesPrenatal Diagnosis · 2026
RNA ‐Based Therapies for Inherited Metabolic DisordersJournal of Inherited Metabolic Disease · 2026