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Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy.
Ann Clin Transl Neurol. 2021 Oct;8(10):1938-1950. doi: 10.1002/acn3.51417. Epub 2021 Aug 28.
Ann Clin Transl Neurol. 2021.
PMID: 34453498
Free PMC article.
Clinical Trial.
Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.
Sellier P, Vidal P, Bertin B, Gicquel E, Bertil-Froidevaux E, Georger C, van Wittenberghe L, Miranda A, Daniele N, Richard I, Gross DA, Mingozzi F, Collaud F, Ronzitti G.
Sellier P, et al.
J Inherit Metab Dis. 2024 Jan;47(1):119-134. doi: 10.1002/jimd.12625. Epub 2023 May 29.
J Inherit Metab Dis. 2024.
PMID: 37204237
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