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Long-term experience in treating patients with spinal muscular atrophy in Poland: from registry to therapies.

MedExpress Team


Published Aug. 31, 2023 12:12

Long-term experience in treating patients with spinal muscular atrophy in Poland: from registry to therapies. - Header image
A long-term study from actual clinical practice extending to a 30-month follow-up of a group of 120 adults and older children with spinal muscular atrophy, with varying disease severity and type of SMA, has shown that patients treated with nusinersen therapy not only experience stabilization of the disease, but in 94 percent there is an improvement in functional status seen over time. In addition, the study shows that the therapy is well tolerated, with 85 percent of patients experiencing improvement and 100 percent achieving stabilization of the condition. The results of the study were published in August this year in the international scientific journal Orphanet Journal of Rare Diseases, in a publication titled: "Long-term follow-up of nusinersen treatment in a wide spectrum of spinal muscular atrophy severity: real-world experience."[1]

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 (≥...

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