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AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial.
Butterfield RJ, Shieh PB, Li H, Binks M, McDonnell TG, Ryan KA, Delnomdedieu M, Belluscio BA, Neelakantan S, Levy DI, Schwartz PF, Smith EC. Butterfield RJ, et al. Among authors: belluscio ba. Nat Med. 2025 Aug;31(8):2712-2721. doi: 10.1038/s41591-025-03750-3. Epub 2025 Jun 27. Nat Med. 2025. PMID: 40579547 Free PMC article. Clinical Trial.
Volumetric MRI-Based Biomarkers in Huntington's Disease: An Evidentiary Review.
Kinnunen KM, Schwarz AJ, Turner EC, Pustina D, Gantman EC, Gordon MF, Joules R, Mullin AP, Scahill RI, Georgiou-Karistianis N; Huntington's Disease Regulatory Science Consortium (HD-RSC). Kinnunen KM, et al. Front Neurol. 2021 Sep 21;12:712555. doi: 10.3389/fneur.2021.712555. eCollection 2021. Front Neurol. 2021. PMID: 34621236 Free PMC article. Review.