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From Genes to Function: Clinical Experience with the Effectiveness and Safety of Risdiplam and Nusinersen in Spinal
Parinaz Sedighi1,2, Afshin Fayyazi3, Firozeh Hosseini3
1Department of Radiology, Imam Khomeini Hospital Complex (IKHC), Tehran University of Medical Sciences, Tehran, Iran.
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.
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