What is the effect of Evrysdi tablets
Evrysdi tablets are an oral medication used to treat spinal muscular atrophy (SMA), which increases functional SMN protein production by regulating SMN2 gene splicing. Its efficacy has been validated in 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 12 months of treatment enabled 41% of children to achieve unsupported sitting and significantly improved motor 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 the natural course of the disease.
2. Response to special groups
(1) SMN2 copy number: Patients with 2 copies have the most significant therapeutic effect, 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 use.
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