Small molecule recognition of disease-relevant RNA structures - Chemical Society Reviews (RSC Publishing) DOI:10.1039/D0CS00560F
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Computational model and schematic of mechanism of action. (a)... | Download Scientific Diagram
Risdiplam: Editing on the RNA level – a dream or a nightmare? – Spinal Muscular Atrophy as example
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Frontiers | Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment | Pharmacology
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Risdiplam - Wikipedia
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Gene specific therapies – the next therapeutic milestone in neurology | SpringerLink
Comparative evaluation of AAV gene therapy, antisense therapy and small molecules therapy for treatment of SMA for efficacy and
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Spinal muscular atrophy: Broad disease spectrum and sex-specific phenotypes - ScienceDirect
Mechanism of action of nusinersen | Download Scientific Diagram
Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines
Risdiplam: Editing on the RNA level – a dream or a nightmare? – Spinal Muscular Atrophy as example
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscula
IJMS | Free Full-Text | New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand? | HTML
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscula
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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 -
The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy
