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Sponsored Artificial Intelligence: A Modern Approach, Global Edition Artificial Intelligence: A Modern Approach, Global Edition

Author

Parinaz Sedighi

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Jun 2026

From Genes to Function: Clinical Experience with the Effectiveness and Safety of Risdiplam and Nusinersen in Spinal Muscular Atrophy

Objective: Spinal muscular atrophy (SMA) involves the survival motor neuron (SMN) 1 gene, leading to motor neuron degeneration. SMN2 is a homologous gene to SMN1, which can produce SMN protein at lower levels. The new gene-based drugs modify SMN2 pre-messenger RNA splicing, leading to production of functional SMN prote...

Parinaz Sedighi, A. Fayyazi, Firozeh Hosseini et al. · 0 citations

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