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EAN 2022 | Using real-world data in SMA management

Anna Kostera-Pruszczyk, MD, PhD, Medical University of Warsaw, Warsaw, Poland, explains how real-world data (RWD) guides the management of spinal muscular atrophy (SMA). Since 2017 three disease-modifying therapies have been approved after clinical trials conducted in pediatric or young-adults. There is an increasing need for RWD on the long-term efficacy and safety of DMTs in SMA patients in patient cohorts reflecting the whole age and severity spectrum. Observational studies show that although most dynamic improvement is seen in pediatric SMA, adult patients, even in severe SMA1 cases, also benefit from treatment, demonstrating improved function. This interview took place at the European Academy of Neurology (EAN) 2022 Congress in Vienna, Austria.


Prof. Anna Kostera-Pruszczyk is an investigator in SMA trials sponsored by F. Hoffmann-La Roche. She has received compensation for participation at symposia, lectures and scientific advisory boards from Biogen, F. Hoffmann-La Roche, AveXis/Novartis and PTC. She also received institutional grant support from Biogen.