| 1 |
33823304 | Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges. | 2022 Jan 5 |
1 |
| 2 |
34515035 | Cytochemical Characterization of Cerebrospinal Fluid Macrophage Inclusions in Pediatric Patients Receiving Intrathecal Nusinersen (SPINRAZA®) for Spinal Muscular Atrophy. | 2022 |
2 |
| 3 |
35088120 | Heat increases full-length SMN splicing: promise for splice-augmenting therapies for SMA. | 2022 Feb |
2 |
| 4 |
35409296 | Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts. | 2022 Apr 1 |
1 |
| 5 |
33074187 | Real-World Data from Nusinersen Treatment for Patients with Later-Onset Spinal Muscular Atrophy: A Single Center Experience. | 2021 |
1 |
| 6 |
33300159 | Identification of SRSF10 as a regulator of SMN2 ISS-N1. | 2021 Mar |
1 |
| 7 |
33459657 | Clinical Outcomes in Patients with Spinal Muscular Atrophy Type 1 Treated with Nusinersen. | 2021 |
1 |
| 8 |
33575118 | Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy. | 2021 Mar 5 |
2 |
| 9 |
33759979 | Managing intrathecal administration of nusinersen in adolescents and adults with 5q-spinal muscular atrophy and previous spinal surgery. | 2021 Feb |
1 |
| 10 |
33997096 | Spinal Muscular Atrophy: The Use of Functional Motor Scales in the Era of Disease-Modifying Treatment. | 2021 Jan-Dec |
1 |
| 11 |
34681015 | Detection of Spinal Muscular Atrophy Patients Using Dried Saliva Spots. | 2021 Oct 14 |
1 |
| 12 |
32056234 | Combinatorial treatment for spinal muscular atrophy: An Editorial for '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' on page 264. | 2020 Apr |
1 |
| 13 |
32392694 | New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand? | 2020 May 7 |
1 |
| 14 |
33060681 | Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy. | 2020 Oct 15 |
1 |
| 15 |
31956255 | Spinal Muscular Atrophy: New Screening System with Real-Time mCOP-PCR and PCR-RFLP for SMN1 Deletion. | 2019 Jul 16 |
1 |
| 16 |
31956256 | Spinal Muscular Atrophy: Advanced Version of Screening System with Real-Time mCOP-PCR and PCR-RFLP for SMN1 Deletion. | 2019 Jul 16 |
1 |
| 17 |
33072999 | A Novel System for Spinal Muscular Atrophy Screening in Newborns: Japanese Pilot Study. | 2019 Dec |
1 |
| 18 |
29294245 | Time Is Motor Neuron: Therapeutic Window and Its Correlation with Pathogenetic Mechanisms in Spinal Muscular Atrophy. | 2018 Aug |
2 |
| 19 |
29380287 | Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy. | 2018 Mar |
1 |
| 20 |
29806836 | Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts. | 2018 May 11 |
1 |
| 21 |
29985170 | Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain. | 2018 Aug 1 |
1 |
| 22 |
30171534 | Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy. | 2018 |
1 |
| 23 |
30414815 | Changing respiratory expectations with the new disease trajectory of nusinersen treated spinal muscular atrophy [SMA] type 1. | 2018 Sep |
1 |
| 24 |
28271991 | New Therapy for Spinal Muscular Atrophy Offers Modest Bang for Pharamaceutical Buck. | 2017 Feb |
1 |
| 25 |
28522225 | Genetic screening of spinal muscular atrophy using a real-time modified COP-PCR technique with dried blood-spot DNA. | 2017 Oct |
1 |
| 26 |
28604635 | Splice-Switching Therapy for Spinal Muscular Atrophy. | 2017 Jun 12 |
1 |
| 27 |
28639617 | Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing. | 2017 Sep |
1 |
| 28 |
28799578 | Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy. | 2017 Jun |
2 |
| 29 |
28977438 | Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape. | 2017 Oct 1 |
1 |
| 30 |
29091570 | Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. | 2017 Nov 2 |
1 |
| 31 |
29434172 | New, Improved Version of the mCOP-PCR Screening System for Detection of Spinal Muscular Atrophy Gene (SMN1) Deletion. | 2017 Sep 7 |
1 |
| 32 |
29434179 | SMA Diagnosis: Detection of SMN1 Deletion with Real-Time mCOP-PCR System Using Fresh Blood DNA. | 2017 Dec 18 |
1 |
| 33 |
27499521 | The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype. | 2016 Sep 1 |
1 |
| 34 |
25727246 | Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions. | 2015 Apr |
1 |
| 35 |
25791416 | SMA screening system using dried blood spots on filter paper: application of COP-PCR to the SMN1 deletion test. | 2015 Jan 19 |
1 |
| 36 |
25920617 | Moving towards treatments for spinal muscular atrophy: hopes and limits. | 2015 Sep |
1 |
| 37 |
26123042 | Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches. | 2015 Jun 30 |
1 |
| 38 |
26381381 | Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy. | 2015 |
1 |
| 39 |
23630626 | Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy. | 2013 |
1 |
| 40 |
23916701 | miR-18b inhibits TGF-β1-induced differentiation of hair follicle stem cells into smooth muscle cells by targeting SMAD2. | 2013 Aug 30 |
1 |
| 41 |
19997596 | Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAs. | 2009 Dec 8 |
2 |
| 42 |
17647030 | Robust quantification of the SMN gene copy number by real-time TaqMan PCR. | 2007 Nov |
2 |
| 43 |
16449646 | Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron. | 2006 Feb |
1 |
| 44 |
11579091 | Thrombin differentiates normal lung fibroblasts to a myofibroblast phenotype via the proteolytically activated receptor-1 and a protein kinase C-dependent pathway. | 2001 Nov 30 |
1 |
| 45 |
11584013 | Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing. | 2001 Nov 30 |
1 |
| 46 |
11769699 | [The Rsa I and Sma I polymorphisms in the von Willebrand factor gene discrimination by hybridization with oligonucleotide arrays]. | 2001 Oct |
1 |