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8. 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; 25(10):1885-1899. PubMed ID: 26931466 [TBL] [Abstract][Full Text] [Related]
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20. The Biochemistry of Survival Motor Neuron Protein Is Paving the Way to Novel Therapies for Spinal Muscle Atrophy. Lomonte P; Baklouti F; Binda O Biochemistry; 2020 Apr; 59(14):1391-1397. PubMed ID: 32227847 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]