How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy | Gene Therapy
Antisense oligonucleotide targeting of SMN2 splicing and SOD1... | Download Scientific Diagram
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
Advances in oligonucleotide drug delivery | Nature Reviews Drug Discovery
Nusinersen (Spinraza®) – Spinal Muscular Atrophy (SMA) | National Institute of Neurological Disorders and Stroke
Frontiers | History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy
Therapeutic strategies for spinal muscular atrophy (SMA). A)... | Download Scientific Diagram
Quality of Life in SMA Patients Under Treatment With Nusinersen
Nusinersen | SMN Inducer | MedChemExpress
The light at the end of the tunnel gets vivid for spinal muscular atrophy - Dutta - 2020 - Journal of Neurochemistry - Wiley Online Library
Cut and Paste: Treating Spinal Muscular Atrophy with Nusinersen - YouTube
Nusinersen - Wikipedia
Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model | Scientific Reports
FN170919 | 1258984-36-9 | Nusinersen | Biosynth
Antisense oligonucleotides (ASOs) in clinical use. (A) Nusinersen binds... | Download Scientific Diagram
CSF and plasma concentrations of nusinersen | Download Scientific Diagram
Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy: Molecular Therapy - Nucleic Acids
Mechanism of approved therapeutics. (A) Nusinersen regulates splicing... | Download Scientific Diagram
Nusinersen - Wikipedia
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
Biomolecules | Free Full-Text | Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy
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 - Pagliarini - 2020 - Journal of Neurochemistry -
Nusinersen - Wikipedia
Spinraza (Nusinersen) for SMA | SMA News Today
Biomolecules | Free Full-Text | Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy
IJMS | Free Full-Text | High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome
