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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
medicine
Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study
Journal of Neuromuscular Diseases, Volume 10, No. 1, Year 2023
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Description
Background and objectives: Disease progression in patients with spinal muscular atrophy (SMA) has changed dramatically within the past years due to the approval of three different disease-modifying treatments. Nusinersen was the first drug to be approved for the treatment of SMA patients. Clinical trials provided data from infants with SMA type 1 and children with SMA type 2, but there is still insufficient evidence and only scarcely reported long-term experience for nusinersen treatment in ambulant patients. Here, we report data from the SMArtCARE registry of ambulant patients under nusinersen treatment with a follow-up period of up to 38 months. Methods: SMArtCARE is a disease-specific registry in Germany, Austria and Switzerland. Data are collected as real-world data during routine patient visits. Our analysis included all patients under treatment with nusinersen able to walk independently before start of treatment with focus on changes in motor function. Results: Data from 231 ambulant patients were included in the analysis. During the observation period, 31 pediatric walkers (27.2%) and 31 adult walkers (26.5%) experienced a clinically meaningful improvement of≥30 m in the 6-Minute-Walk-Test. In contrast, only five adult walkers (7.7%) showed a decline in walking distance≥30 m, and two pediatric walkers (1.8%) lost the ability to walk unassisted under treatment with nusinersen. HFMSE and RULM scores improved in pediatric and remained stable in adult patients. Conclusion: Our data demonstrate a positive effect of nusinersen treatment in most ambulant pediatric and adult SMA patients. We not only observed a stabilization of disease progression or lack of deterioration, but clinically meaningful improvements in walking distance. © 2023 - The authors. Published by IOS Press.
Authors & Co-Authors
Hagenacker, Tim
Germany, Essen
Medizinischen Fakultät
Walter, Maggie Christine
Germany, Munich
Ludwig-maximilians-universität München
Blaschek, Astrid
Germany, Munich
Ludwig-maximilians-universität München
Baumann, Matthias
Austria, Innsbruck
Medizinische Universitat Innsbruck
Baumgartner, Manuela
Unknown Affiliation
Hahn, Andreas
Germany, Giessen
Justus-liebig-universität Gießen
Küpper, Hanna
Germany, Tubingen
Universitätsklinikum Und Medizinische Fakultät Tübingen
Johannsen, Jessika
Germany, Hamburg
Universitätsklinikum Hamburg-eppendorf
Kolbel, Heike
Germany, Duisburg
Universität Duisburg-essen
Schrank, Bertold
Germany, Wiesbaden
Dkd Helios Klinik Wiesbaden
Schreiber, Gudrun
Germany, Kassel
Klinikum Kassel Gmbh
Smitka, Martin
Germany, Dresden
Technische Universität Dresden
Ziegler, Andreas E.
Germany, Heidelberg
Universitätsklinikum Heidelberg
Lochmüller, Hanns
Germany, Freiburg Im Breisgau
Universität Freiburg
Canada, Ottawa
Children's Hospital of Eastern Ontario, Ottawa
Canada, Ottawa
L'hôpital D'ottawa
Canada, Ottawa
University of Ottawa
Kirschner, Janbernd
Germany, Freiburg Im Breisgau
Universität Freiburg
Statistics
Citations: 6
Authors: 15
Affiliations: 35
Identifiers
Doi:
10.3233/JND-221600
ISSN:
22143599
Research Areas
Health System And Policy
Maternal And Child Health
Study Design
Cohort Study