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Journal Abstract Search

676 related items for PubMed ID: 30553700

  • 1. The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy.
    Kletzl H, Marquet A, Günther A, Tang W, Heuberger J, Groeneveld GJ, Birkhoff W, Mercuri E, Lochmüller H, Wood C, Fischer D, Gerlach I, Heinig K, Bugawan T, Dziadek S, Kinch R, Czech C, Khwaja O.
    Neuromuscul Disord; 2019 Jan; 29(1):21-29. PubMed ID: 30553700
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  • 3. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
    Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, Petruska J, Narasimhan J, Dakka A, Welch EM, Karp G, Chen KS, Metzger F, Ratni H, Lotti F, Tisdale S, Naryshkin NA, Pellizzoni L, Paushkin S, Ko CP, Weetall M.
    Hum Mol Genet; 2016 May 15; 25(10):1885-1899. PubMed ID: 26931466
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  • 7. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
    Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F.
    Science; 2014 Aug 08; 345(6197):688-93. PubMed ID: 25104390
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  • 8. Securinine enhances SMN2 exon 7 inclusion in spinal muscular atrophy cells.
    Chen YC, Chang JG, Liu TY, Jong YJ, Cheng WL, Yuo CY.
    Biomed Pharmacother; 2017 Apr 08; 88():708-714. PubMed ID: 28152480
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  • 10. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
    Poirier A, Weetall M, Heinig K, Bucheli F, Schoenlein K, Alsenz J, Bassett S, Ullah M, Senn C, Ratni H, Naryshkin N, Paushkin S, Mueller L.
    Pharmacol Res Perspect; 2018 Dec 08; 6(6):e00447. PubMed ID: 30519476
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  • 11. Effects of Inhibitors of SLC9A-Type Sodium-Proton Exchangers on Survival Motor Neuron 2 (SMN2) mRNA Splicing and Expression.
    Kanda S, Moulton E, Butchbach MER.
    Mol Pharmacol; 2022 Aug 08; 102(2):92-105. PubMed ID: 35667685
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  • 12. Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells.
    Pagliarini V, Guerra M, Di Rosa V, Compagnucci C, Sette C.
    J Neurochem; 2020 Apr 08; 153(2):264-275. PubMed ID: 31811660
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  • 14. Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy.
    Ando S, Suzuki S, Okubo S, Ohuchi K, Takahashi K, Nakamura S, Shimazawa M, Fuji K, Hara H.
    Sci Rep; 2020 Oct 15; 10(1):17472. PubMed ID: 33060681
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  • 16. ZPR1 prevents R-loop accumulation, upregulates SMN2 expression and rescues spinal muscular atrophy.
    Kannan A, Jiang X, He L, Ahmad S, Gangwani L.
    Brain; 2020 Jan 01; 143(1):69-93. PubMed ID: 31828288
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  • 17. Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy.
    Thurmond J, Butchbach ME, Palomo M, Pease B, Rao M, Bedell L, Keyvan M, Pai G, Mishra R, Haraldsson M, Andresson T, Bragason G, Thosteinsdottir M, Bjornsson JM, Coovert DD, Burghes AH, Gurney ME, Singh J.
    J Med Chem; 2008 Feb 14; 51(3):449-69. PubMed ID: 18205293
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  • 19. Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.
    Hua Y, Sahashi K, Rigo F, Hung G, Horev G, Bennett CF, Krainer AR.
    Nature; 2011 Oct 05; 478(7367):123-6. PubMed ID: 21979052
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  • 20. Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice.
    Kray KM, McGovern VL, Chugh D, Arnold WD, Burghes AHM.
    Neurobiol Dis; 2021 Nov 05; 159():105488. PubMed ID: 34425216
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