These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
30. An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy. Li Y; Zeng H; Wei Y; Ma X; He Z Hum Gene Ther; 2023 Mar; 34(5-6):180-191. PubMed ID: 36762938 [TBL] [Abstract][Full Text] [Related]
31. Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA. Leckie J; Yokota T Molecules; 2024 Jun; 29(11):. PubMed ID: 38893532 [TBL] [Abstract][Full Text] [Related]
32. Nusinersen: A Review in 5q Spinal Muscular Atrophy. Hoy SM CNS Drugs; 2018 Jul; 32(7):689-696. PubMed ID: 30027400 [TBL] [Abstract][Full Text] [Related]
33. Cell-Based Therapy for Spinal Muscular Atrophy. Han F; Ebrahimi-Barough S; Abolghasemi R; Ai J; Liu Y Adv Exp Med Biol; 2020; 1266():117-125. PubMed ID: 33105498 [TBL] [Abstract][Full Text] [Related]
34. New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand? Chen TH Int J Mol Sci; 2020 May; 21(9):. PubMed ID: 32392694 [TBL] [Abstract][Full Text] [Related]
35. 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; 10(1):17472. PubMed ID: 33060681 [TBL] [Abstract][Full Text] [Related]
36. High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed Wijaya YOS; Niba ETE; Nishio H; Okamoto K; Awano H; Saito T; Takeshima Y; Shinohara M Genes (Basel); 2022 Apr; 13(4):. PubMed ID: 35456491 [TBL] [Abstract][Full Text] [Related]
37. Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene. Pao PW; Wee KB; Yee WC; Pramono ZA Mol Ther; 2014 Apr; 22(4):854-61. PubMed ID: 24317636 [TBL] [Abstract][Full Text] [Related]
38. A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes. Singh NN; Seo J; Rahn SJ; Singh RN PLoS One; 2012; 7(11):e49595. PubMed ID: 23185376 [TBL] [Abstract][Full Text] [Related]
39. Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts. Farrelly-Rosch A; Lau CL; Patil N; Turner BJ; Shabanpoor F Neurochem Int; 2017 Sep; 108():213-221. PubMed ID: 28389270 [TBL] [Abstract][Full Text] [Related]