19 results on '"Ratni, Hasane"'
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2. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression
3. Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines
4. Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
5. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
6. Discovery of RO7185876, a Highly Potent γ-Secretase Modulator (GSM) as a Potential Treatment for Alzheimer’s Disease
7. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs
8. A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier
9. Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
10. Diversity oriented biosynthesis via accelerated evolution of modular gene clusters
11. A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.
12. Pharmacokinetics, pharmacodynamics, and efficacy of a small-moleculeSMN2splicing modifier in mouse models of spinal muscular atrophy
13. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
14. From Planar Chiral o-Chloro- and o-Iodo Benzaldehyde Tricarbonyl Chromium Complexes to Enantiopure Fused Hydroisoquinolines and Hydroquinolines
15. Arene Chromium Complexes: Heterocyclic Chiral Auxiliaries and Synthetic Targets
16. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
17. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
18. Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
19. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
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