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Therapeutic developments for valosin-containing protein mediated multisystem proteinopathy.
Curr Opin Neurol. 2023 Oct 1;36(5):432-440. doi: 10.1097/WCO.0000000000001184. Epub 2023 Aug 30.
Curr Opin Neurol. 2023.
PMID: 37678339
Review.
Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease.
Weiss L, Carrer M, Shmara A, Cheng C, Yin H, Ta L, Boock V, Fazeli Y, Chang M, Paguio M, Lee J, Yu H, Martin A, Raben N, Weiss J, Grossman T, Jafar-Nejad P, Kimonis V.
Weiss L, et al. Among authors: boock v.
bioRxiv [Preprint]. 2024 Mar 2:2024.02.22.580414. doi: 10.1101/2024.02.22.580414.
bioRxiv. 2024.
PMID: 38464319
Free PMC article.
Preprint.
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