Spinal Muscular Atrophy disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
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
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
Frontiers | Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries | Pharmacology
DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy - ScienceDirect
Spinale musculaire atrofie - Wikipedia
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
Spinal muscular atrophy (sma)
Oligonucleotide therapeutics: How to measure plasma concentration, tissue distribution and immunogenicity?
Spinal Muscular Atrophy - 1st Edition
Risdiplam - an overview | ScienceDirect Topics
Therapeutic approaches for spinal muscular atrophy (SMA) | Gene Therapy
The Challenges of Determining the Safety and Efficacy of Risdiplam in SMA - Rare Disease Advisor
Constantin d'Ydewalle - Senior Scientist, Neuroscience Drug Discovery - The Janssen Pharmaceutical Companies of Johnson & Johnson | LinkedIn
Spinal muscular atrophy (sma)
Nutrients | Free Full-Text | Nutritional, Gastrointestinal and Endo-Metabolic Challenges in the Management of Children with Spinal Muscular Atrophy Type 1 | HTML
Cost-effectiveness clouds for nusinersen versus BSC | Download Scientific Diagram
Nusinersen expanded access program (EAP) enrollment. The nusinersen EAP... | Download Scientific Diagram
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
Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy | Orphanet Journal of Rare Diseases | Full Text
Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review | SpringerLink
