From Genes to Function: Clinical Experience with the Effectiveness and Safety of Risdiplam and Nusinersen in Spinal Muscular Atrophy
Iranian Journal of Child Neurology,
Vol. 20 No. 3 (2026),
1 June 2026
,
Page 55-61
https://doi.org/10.22037/ijcn.v20i3.51971
Abstract
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 protein.
Materials & Methods:
This study aimed to evaluate the effectiveness and safety of Risdiplam and Nusinersen in patients with SMA types I–III. Hammersmith Functional Motor Scale-Expanded (HFMSE) was used for motor evaluation.
Results:
Results revealed that changes were significant after six months (p< 0.001). Improvements were compared between the two drugs, age groups, and SMA disease types, and no significant differences were found. No severe side effects were experienced and only a few patients reported headaches, and backaches following Nusinersen.
Conclusion:
both Risdiplam and Nusinersen led to significant improvements in motor function; however, based on cost-effectiveness considerations, we recommend Risdiplam.
- Acute Spinal Subdural Hematoma
- risdiplam
- nusinersen
- Genetic Diseases
How to Cite
References
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