Not just stabilization, but increasingly visible improvement
- Our published results of a long follow-up of 30 months of adult patients with SMA showed, in the form of scores on the relevant scales, what we observe every day, i.e. an improvement in functional status that becomes more pronounced over time," says Prof. Anna Kostera-Pruszczyk, co-author of the study, head of the Department of Neurology at UCK WUM, ERN EURO NMD Expert Center for Rare Neuromuscular Diseases.
- These are very positive results, especially for adults who start treatment after decades of illness, often with very severe paresis. In the study, we showed that in the group of 120 patients we treated, not only did the disease stabilize, but with each successive administration of the drug we saw a slightly greater improvement than the previous one, the expert adds.
After 30 months of follow-up, an increase of 5.1 points was observed in the HFMSE scale, and an increase of 5.59 points in the CHOP-INTEND test (at 26 months of follow-up). The average RULM score improved by 1.96 points at 30 months.
At 30 months of treatment, respectively, clinically significant improvements in the HFMSE scale (≥...