16 results on '"Dakka, Amal"'
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2. A deep learning approach to identify gene targets of a therapeutic for human splicing disorders
3. Small molecule splicing modifiers with systemic HTT-lowering activity
4. Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
5. Development of a novel oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia
6. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
7. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression
8. MOTOR NEURON DISEASE: SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
9. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
10. A deep learning approach to identify new gene targets of a novel therapeutic for human splicing disorders
11. ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia
12. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy
13. Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy
14. Pharmacokinetics, pharmacodynamics, and efficacy of a small-moleculeSMN2splicing modifier in mouse models of spinal muscular atrophy
15. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
16. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
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