How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy | Gene Therapy
Attachment: Product Information: Nusinersen (as heptadecasodium)
Splicing and the SMN2 'Back-Up' Gene - Spinal Muscular Atrophy UK
Splicing and the SMN2 'Back-Up' Gene - Spinal Muscular Atrophy UK
Nusinersen - Wikipedia
The Discovery of Spinraza - Cure SMA
Frontiers | History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy
Spinraza: Package Insert - Drugs.com
Spinraza – TreatSMA
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
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text
SPINRAZA (nusinersen) injection, for intrathecal use
Frontiers | History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy
LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts. - Abstract - Europe PMC
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
Spinraza, INN - nusinersen
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text
Glen Report 32.14: New Product — 2'-MOE RNA Phosphoramidites
How SPINRAZA® (nusinersen) Works | HCP
Mechanism of action of nusinersen | Download Scientific Diagram
nusinersen | Ligand page | IUPHAR/BPS Guide to PHARMACOLOGY
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
