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9 Publications listed
Exploratory Project Report: Individualised therapies and platform technologies: exploring methodological challenges for NICE project report
Published 2025
University of Oxford
Bias in HD-ISS staging introduced by the FreeSurfer cross-sectional stream: Insights from the Huntington's Disease Young Adult Study (HD-YAS)
Published 2025
Huntington's disease (HD)
Brain
HTT
University College London
DOI: 10.1177/18796397251366900. Epub 2025 Aug 13.
Citation: J Huntingtons Dis. 2025 Nov;14(4):396-402.
Huntington disease: somatic expansion, pathobiology and therapeutics
Published 2025
Huntington's disease (HD)
Brain
HTT
University College London
DOI: 10.1038/s41582-025-01159-7. Online ahead of print.
Citation: Nat Rev Neurol. 2025 Nov 13.
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy
Published 2025
Spinal Muscular Atrophy (SMA)
University of Oxford
DOI: 10.1080/14737175.2025.2579903. Online ahead of print.
Citation: Expert Rev Neurother 2025 Nov 13:1-20.
Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry
Published 2025
Spinal Muscular Atrophy (SMA)
University of Oxford
DOI: 10.1177/22143602251395173. Online ahead of print.
Citation: J Neuromuscul Dis. 2025 Nov 11:22143602251395173.