Spinal muscular atrophy (SMA) is an inherited muscle disease. Current treatments for SMA have been shown to significantly improve the course of the disease, but long-term outcome data remains scarce. This study looked at patients registered in the Swiss-Reg-NMD with SMA type 1–3, who are treated with nusinersen over a period of at least four years (4.1–6.2 years). The study analyzed how well motor function developed, if patients need support to eat or breathe and how patients feel about the effect of the treatment themselves (PROMS).
Our data confirms that nusinersen treatment stabilizes or even improves motor function in SMA. The patients who started their treatment within the first year of their symptoms were the ones who showed the most improvement. However, patients who started the treatment later showed also improvements in our study group. After four years, patients still said that the treatment is beneficial and barely anyone in the group felt worse over this period.
The article has been published here: Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy