| 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 |
1 |
| 3 |
35088120 | Heat increases full-length SMN splicing: promise for splice-augmenting therapies for SMA. | 2022 Feb |
1 |
| 4 |
35159227 | Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy. | 2022 Jan 26 |
2 |
| 5 |
35409296 | Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts. | 2022 Apr 1 |
1 |
| 6 |
35456491 | High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed SMN2 Splicing in Patient Fibroblasts. | 2022 Apr 13 |
1 |
| 7 |
33300159 | Identification of SRSF10 as a regulator of SMN2 ISS-N1. | 2021 Mar |
2 |
| 8 |
33531827 | Spinal Muscular Atrophy: Mutations, Testing, and Clinical Relevance. | 2021 |
1 |
| 9 |
33575118 | Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy. | 2021 Mar 5 |
2 |
| 10 |
33997096 | Spinal Muscular Atrophy: The Use of Functional Motor Scales in the Era of Disease-Modifying Treatment. | 2021 Jan-Dec |
2 |
| 11 |
34231920 | Selecting disease-modifying medications in 5q spinal muscular atrophy. | 2021 Oct |
1 |
| 12 |
34445083 | High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome. | 2021 Aug 4 |
2 |
| 13 |
34721262 | Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular Atrophy. | 2021 |
1 |
| 14 |
34884240 | Combination Therapy with Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I. | 2021 Nov 26 |
1 |
| 15 |
31811660 | 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. | 2020 Apr |
2 |
| 16 |
31909520 | Drosophila SMN2 minigene reporter model identifies moxifloxacin as a candidate therapy for SMA. | 2020 Feb |
1 |
| 17 |
31922583 | Spinal muscular atrophy - new therapies, new challenges. | 2020 |
1 |
| 18 |
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 |
| 19 |
32233169 | Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy. | 2020 Apr |
1 |
| 20 |
32294764 | Targeted Treatments for Inherited Neuromuscular Diseases of Childhood. | 2020 Jun |
1 |
| 21 |
32784322 | Successful Combination of Neuraxial and Regional Anesthesia in a Child With Advanced Spinal Muscular Atrophy Type 1 Receiving Maintenance Nusinersen Therapy: A Case Report. | 2020 Apr |
1 |
| 22 |
33060681 | Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy. | 2020 Oct 15 |
1 |
| 23 |
33105498 | Cell-Based Therapy for Spinal Muscular Atrophy. | 2020 |
1 |
| 24 |
33283185 | The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy. | 2020 |
1 |
| 25 |
30221755 | Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps. | 2019 Jan |
1 |
| 26 |
31425176 | Therapeutic advances in SMA. | 2019 Oct |
1 |
| 27 |
31956255 | Spinal Muscular Atrophy: New Screening System with Real-Time mCOP-PCR and PCR-RFLP for SMN1 Deletion. | 2019 Jul 16 |
1 |
| 28 |
29380287 | Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy. | 2018 Mar |
1 |
| 29 |
29434670 | Advances in spinal muscular atrophy therapeutics. | 2018 |
1 |
| 30 |
29443664 | Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. | 2018 Feb 15 |
2 |
| 31 |
29742241 | Therapeutic advances in 5q-linked spinal muscular atrophy. | 2018 Apr |
1 |
| 32 |
29806836 | Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts. | 2018 May 11 |
1 |
| 33 |
29858094 | Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript. | 2018 Jun 1 |
2 |
| 34 |
29916015 | Mechanism of Splicing Regulation of Spinal Muscular Atrophy Genes. | 2018 |
1 |
| 35 |
28193854 | Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy. | 2017 Feb 21 |
1 |
| 36 |
28229309 | Nusinersen: First Global Approval. | 2017 Mar |
1 |
| 37 |
28271991 | New Therapy for Spinal Muscular Atrophy Offers Modest Bang for Pharamaceutical Buck. | 2017 Feb |
1 |
| 38 |
28400976 | ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy. | 2017 Jan |
1 |
| 39 |
28522225 | Genetic screening of spinal muscular atrophy using a real-time modified COP-PCR technique with dried blood-spot DNA. | 2017 Oct |
1 |
| 40 |
28561813 | Therapeutic approaches for spinal muscular atrophy (SMA). | 2017 Sep |
1 |
| 41 |
28623256 | LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts. | 2017 Jun 16 |
2 |
| 42 |
28639617 | Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing. | 2017 Sep |
1 |
| 43 |
28768735 | Therapeutic strategies for spinal muscular atrophy: SMN and beyond. | 2017 Aug 1 |
1 |
| 44 |
28799578 | Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy. | 2017 Jun |
1 |
| 45 |
28983837 | Treatment Advances in Spinal Muscular Atrophy. | 2017 Oct 6 |
1 |
| 46 |
29091570 | Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. | 2017 Nov 2 |
1 |
| 47 |
25727246 | Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions. | 2015 Apr |
1 |
| 48 |
25791416 | SMA screening system using dried blood spots on filter paper: application of COP-PCR to the SMN1 deletion test. | 2015 Jan 19 |
1 |
| 49 |
25920617 | Moving towards treatments for spinal muscular atrophy: hopes and limits. | 2015 Sep |
2 |
| 50 |
26123042 | Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches. | 2015 Jun 30 |
1 |