![Medicina | Free Full-Text | The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials Medicina | Free Full-Text | The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials](https://www.mdpi.com/medicina/medicina-58-00213/article_deploy/html/images/medicina-58-00213-g001.png)
Medicina | Free Full-Text | The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials
![IJMS | Free Full-Text | Nusinersen Modulates Proteomics Profiles of Cerebrospinal Fluid in Spinal Muscular Atrophy Type 1 Patients IJMS | Free Full-Text | Nusinersen Modulates Proteomics Profiles of Cerebrospinal Fluid in Spinal Muscular Atrophy Type 1 Patients](https://pub.mdpi-res.com/ijms/ijms-22-04329/article_deploy/html/images/ijms-22-04329-ag.png?1619083406)
IJMS | Free Full-Text | Nusinersen Modulates Proteomics Profiles of Cerebrospinal Fluid in Spinal Muscular Atrophy Type 1 Patients
Antisense oligonucleotides (ASOs) in clinical use. (A) Nusinersen binds... | Download Scientific Diagram
![Nusinersen (Spinraza®) – Spinal Muscular Atrophy (SMA) | National Institute of Neurological Disorders and Stroke Nusinersen (Spinraza®) – Spinal Muscular Atrophy (SMA) | National Institute of Neurological Disorders and Stroke](https://www.ninds.nih.gov/sites/default/files/styles/full_width_small/public/2022-03/smn2_rna_replacement_v2.png?itok=cHY6Rb7l)
Nusinersen (Spinraza®) – Spinal Muscular Atrophy (SMA) | National Institute of Neurological Disorders and Stroke
![Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text](https://media.springernature.com/lw685/springer-static/image/art%3A10.1186%2Fs42466-021-00162-9/MediaObjects/42466_2021_162_Fig1_HTML.png)
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text
![How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy | Gene Therapy How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy | Gene Therapy](https://media.springernature.com/full/springer-static/image/art%3A10.1038%2Fgt.2017.34/MediaObjects/41434_2017_Article_BFgt201734_Fig1_HTML.jpg)
How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy | Gene Therapy
![Biomolecules | Free Full-Text | Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy Biomolecules | Free Full-Text | Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy](https://www.mdpi.com/biomolecules/biomolecules-12-01431/article_deploy/html/images/biomolecules-12-01431-g002-550.jpg)
Biomolecules | Free Full-Text | Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy
![Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model | Scientific Reports Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model | Scientific Reports](https://media.springernature.com/lw685/springer-static/image/art%3A10.1038%2Fs41598-020-67569-3/MediaObjects/41598_2020_67569_Fig1_HTML.png)
Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model | Scientific Reports
![Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study - The Lancet Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study - The Lancet](https://www.thelancet.com/cms/attachment/28f5d00c-e8d7-4ada-ae38-18dbdaed715e/gr1_lrg.jpg)
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study - The Lancet
![IJMS | Free Full-Text | Development of the Method for Nusinersen and Its Metabolites Identification in the Serum Samples of Children Treated with Spinraza for Spinal Muscular Atrophy IJMS | Free Full-Text | Development of the Method for Nusinersen and Its Metabolites Identification in the Serum Samples of Children Treated with Spinraza for Spinal Muscular Atrophy](https://www.mdpi.com/ijms/ijms-23-10166/article_deploy/html/images/ijms-23-10166-g004.png)
IJMS | Free Full-Text | Development of the Method for Nusinersen and Its Metabolites Identification in the Serum Samples of Children Treated with Spinraza for Spinal Muscular Atrophy
![Biomolecules | Free Full-Text | Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy Biomolecules | Free Full-Text | Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy](https://www.mdpi.com/biomolecules/biomolecules-12-01431/article_deploy/html/images/biomolecules-12-01431-g001-550.jpg)