36 results on '"Ratni, Hasane"'
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2. Development and Optimization of the Manufacturing Process for RNA-Splicing Modifier Risdiplam RG7916
3. Structural basis of a small molecule targeting RNA for a specific splicing correction
4. Rewriting the (tran)script: Application to spinal muscular atrophy
5. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
6. Contribution to the Discovery of a Novel Medicine for a Neuromuscular Disease and of other Promising Molecules for the Treatment of Neurodevelopmental and Neurodegenerative Diseases
7. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression
8. Mapping the binding pocket of a novel, high-affinity, slow dissociating tachykinin NK3 receptor antagonist: Biochemical and electrophysiological characterization
9. MOTOR NEURON DISEASE: SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
10. Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines
11. Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
12. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
13. Discovery of RO7185876, a Highly Potent γ-Secretase Modulator (GSM) as a Potential Treatment for Alzheimer’s Disease
14. Discovery of Balovaptan, a Vasopressin 1a Receptor Antagonist for the Treatment of Autism Spectrum Disorder
15. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs
16. A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier
17. Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
18. Relationship Between Central and Peripheral SMN Protein Increase Upon Treatment with RO7034067 (RG7916) (S46.007)
19. Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
20. Diversity oriented biosynthesis via accelerated evolution of modular gene clusters
21. Oral SMN2 splicing modifiers in spinal muscular atrophy: Proof-of-mechanism and ongoing clinical studies
22. Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy
23. A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.
24. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy
25. Pharmacokinetics, pharmacodynamics, and efficacy of a small-moleculeSMN2splicing modifier in mouse models of spinal muscular atrophy
26. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
27. Discovery of Highly Selective Brain-Penetrant Vasopressin 1a Antagonists for the Potential Treatment of Autism via a Chemogenomic and Scaffold Hopping Approach
28. From Planar Chiral o-Chloro- and o-Iodo Benzaldehyde Tricarbonyl Chromium Complexes to Enantiopure Fused Hydroisoquinolines and Hydroquinolines
29. Arene Chromium Complexes: Heterocyclic Chiral Auxiliaries and Synthetic Targets
30. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
31. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
32. Identification of a Critical Residue in the Transmembrane Domain 2 of Tachykinin Neurokinin 3 Receptor Affecting the Dissociation Kinetics and Antagonism Mode of Osanetant (SR 142801) and Piperidine-Based Structures
33. MAPPING THE BINDING SITE FOR TACHYKININ NK3 RECEPTOR ANTAGONISTS
34. Me-Talnetant and Osanetant Interact within Overlapping but Not Identical Binding Pockets in the Human Tachykinin Neurokinin 3 Receptor Transmembrane Domains
35. Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
36. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
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