Frontiers | Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries | Pharmacology
High-throughput genetic newborn screening for spinal muscular atrophy by rapid nucleic acid extraction from dried blood spots and 384-well qPCR | European Journal of Human Genetics
Nusinersen expanded access program (EAP) enrollment. The nusinersen EAP... | Download Scientific Diagram
Nutrients | Free Full-Text | Nutritional, Gastrointestinal and Endo-Metabolic Challenges in the Management of Children with Spinal Muscular Atrophy Type 1 | HTML
HTA | BeNeLuxA
Spinal Muscular Atrophy - 1st Edition
Spinal muscular atrophy (sma)
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
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 -
Cost-effectiveness clouds for nusinersen versus BSC | Download Scientific Diagram
Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy | Orphanet Journal of Rare Diseases | Full Text
Risdiplam - an overview | ScienceDirect Topics
Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges - IOS Press
Nutrients | Free Full-Text | Nutritional, Gastrointestinal and Endo-Metabolic Challenges in the Management of Children with Spinal Muscular Atrophy Type 1 | HTML
Frontiers | Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries | Pharmacology
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
Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review | SpringerLink
Constantin d'Ydewalle - Senior Scientist, Neuroscience Drug Discovery - The Janssen Pharmaceutical Companies of Johnson & Johnson | LinkedIn
An ASO modification that enhances nuclease resistance, lowers toxicity, and increases binding affinity
Frontiers | Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries | Pharmacology
Newborn screening for SMA in Southern Belgium - Neuromuscular Disorders
Spinal Muscular Atrophy disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
Therapeutic approaches for spinal muscular atrophy (SMA) | Gene Therapy
Nusinersen sodium, ヌシネルセンナトリウム | New Drug Approvals
Cost-effectiveness clouds for nusinersen versus BSC | Download Scientific Diagram
The Challenges of Determining the Safety and Efficacy of Risdiplam in SMA - Rare Disease Advisor
