The 30-month results of a Polish observational study in adults, sicker and older people with SMA, which you co-authored and which was published in the Orphanet Journal of Diseases this August, show not only stabilization of the disease, but also a 94 percent improvement in functional status. Did the state expect such high treatment efficacy with nusinersen therapy when starting the study?
Nusinersen therapy is, of course, a breakthrough in the treatment of spinal muscular atrophy, and of course clinical trials have shown it to be an effective treatment, but primarily in children. The data from the actual clinical practice clinic even exceeded our expectations in some ways, because we expected stabilization in the first place....
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