An ASO modification that enhances nuclease resistance, lowers toxicity, and increases binding affinity
Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges - IOS Press
Nusinersen expanded access program (EAP) enrollment. The nusinersen EAP... | Download Scientific Diagram
Frontiers | Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries | Pharmacology
Nusinersen expanded access program (EAP) enrollment. The nusinersen EAP... | Download Scientific Diagram
Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy | Orphanet Journal of Rare Diseases | Full Text
Understanding European patient expectations towards current therapeutic development in spinal muscular atrophy - Neuromuscular Disorders
Examining the impact of different country processes for appraising rare disease treatments: a case study analysis | International Journal of Technology Assessment in Health Care | Cambridge Core
Nusinersen - Wikipedia
Frontiers | Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries | Pharmacology
Nutrients | Free Full-Text | Nutritional, Gastrointestinal and Endo-Metabolic Challenges in the Management of Children with Spinal Muscular Atrophy Type 1 | HTML
Nutrients | Free Full-Text | Nutritional, Gastrointestinal and Endo-Metabolic Challenges in the Management of Children with Spinal Muscular Atrophy Type 1 | HTML
Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium | Scientific Reports
Spinale musculaire atrofie - Wikipedia
Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4 | Request PDF
Frontiers | Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries | Pharmacology
Cost-effectiveness clouds for nusinersen versus BSC | Download Scientific Diagram
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 sodium, ヌシネルセンナトリウム | New Drug Approvals
Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review | SpringerLink
Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial - The Lancet Neurology
Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy | Orphanet Journal of Rare Diseases | Full Text
