What is the effect of Evrysdi tablets
Evrysdi tablets, as an oral medication for the treatment of spinal muscular atrophy (SMA), exert their effects by regulating SMN2 gene splicing, and their efficacy has been validated in multiple clinical trials. The following provides specific explanations from three aspects: core efficacy, response to special populations, and long-term treatment advantages.
1. Core efficacy data
(1) The infantile SMA: FIREISH study showed that 41% of children achieved unsupported independent sitting after 12 months of treatment, significantly improving motor function milestones.
(2) Delayed onset SMA: The SUNFISH trial confirmed that 24 months of treatment resulted in an average improvement of 1.36 points in patients' MFM32 scale scores, which was better than natural course progression.
2. Response to special groups
(1) SMN2 copy number: Patients with 2 copies have the most significant improvement in motor function, while those with 3 copies need to extend the observation period to 18 months to evaluate the effect.
(2) Age factor: Children under 2 years old show more significant improvement in motor function, while adult patients prioritize stable condition as the main treatment goal.
3 Long term treatment advantages
(1) Continuous benefits: Open label extension trials have shown that 60 months of treatment maintained or further improved motor function in 85% of patients.
(2) Safety: The most common adverse reactions are fever/diarrhea/rash, and the incidence of severe liver/kidney toxicity is less than 1%, making it suitable for long-term medication management.
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