The Orphanet Journal of Rare Diseases published in August this year the first Polish real-world practice data for adult and elderly SMA patients treated with nusinersen. How large a group of patients do these observations apply to and what are the results?
This is a study that my team, that is, the team from the Department of Neurology at the Warsaw Medical University, carried out together with a team led by Dr. Ryszard Nowak from the Rydygier Hospital in Krakow. We treated a total of 130 patients, 120 were included in the analysis. The most important element was the fact of evaluation after at least 6 months of treatment. In fact, a very large group of these patients were observed by us and also evaluated in appropriately selected scales, for a period of time, reaching 30 months. There were no criteria narrowing access to treatment, since the nusinersen treatment program in Poland allows us to treat patients with all types of the disease, with all stages of spinal muscular atrophy. The long follow-up allowed us to draw very positive and, I think, very interesting conclusions, also for neurologists from other centers around the world.
What is behind the terms "positive" and "interesting"?
The first publications from Germany or Italy showed improvements that marked themselves b...