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44 Publications listed
Beyond innovation: why rare disease gene therapy needs a reset
Published 2026
Genomics,
Blood,
Brain,
Eye,
Delivery
Nuffield Department of Clinical Neuroscience, University of Oxford
doi.org/10.1080/14712598.2026.2706035
Abdalla Elsayed, M. E., & MacLaren, R. E. (2026). Beyond innovation: why rare disease gene therapy needs a reset. Expert Opinion on Biological Therapy, 26(7), 651–654. https://doi.org/10.1080/14712598.2026.2706035
EQ-5D vision bolt-on in macular degeneration: associations with visual measures and effect on utility differences and cost-effectiveness of stereotactic radiotherapy
Published 2026
Eye
University of Oxford,
King's College London
doi.org/10.1186/s12955-025-02457-w
Zhu, X., Wordsworth, S., Lee, C.N. et al. Health Qual Life Outcomes 24, 93 (2026). https://doi.org/10.1186/s12955-025-02457-w
Genome-wide association studies of infant and toddler temperament in European and multi-ancestry populations
Published 2026
Genomics
Birkbeck University of London,
University of Surrey,
University College London (UCL),
University of Rome Tor Vergata,
Institute of Developmental and Regenerative Medicine, Department of Paediatrics, University of Oxford,
Lovisenberg Diaconal Hospital, Oslo,
Norwegian Institute of Public Health,
University of Bristol,
Vrije Universiteit Amsterdam,
Amsterdam Public Health Research Institute,
Norwegian Institute of Public Health
10.1038/s41562-026-02486-5
Hollowell A, Gui A, Wigdor E, Morgan MJ, Hannigan LJ, Corfield EC, Pool R, Bruins S, Ask H, Middeldorp CM, Pourcain BS, Bartels M, et al., Nat Hum Behav. 2026 Jul 1. doi: 10.1038/s41562-026-02486-5. Epub ahead of print. PMID: 42386913.
Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial
Published 2026
Brain
Medical University of Gdańsk,
University of Oxford,
University College London (UCL)
10.1016/s2352-4642(26)00126-4
Mazurkiewicz-Bełdzińska M, Servais L, Baranello G et al., The Lancet Child & Adolescent Health, 2026; 10, 625-636
Harmonizing standards and resources for the medical genome
Published 2026
Genomics
Stanford University,
Google, Mountain View,
Sixth Street, San Francisco,
University of California, Berkeley - IGI
doi.org/10.1038/s41586-026-10621-5
Ashley, E.A., Alizadeh, A.A., Armitage, H. et al. Harmonizing standards and resources for the medical genome. Nature 655, 47–58 (2026). https://doi.org/10.1038/s41586-026-10621-5