Spinale Muskelatrophie – Expertenempfehlungen zur Behandlung von erwachsenen Patienten mit Nusinersen

Research output: Contribution to journalResearch articleContributedpeer-review

Contributors

  • Tim Hagenacker - , University Hospital Essen (Author)
  • Andreas Hermann - , Rostock University Medical Centre (Author)
  • Christoph Kamm - , Rostock University Medical Centre (Author)
  • Maggie C Walter - , Hospital of the Ludwig-Maximilians-University (LMU) Munich (Author)
  • Markus Weiler - , University Hospital Heidelberg (Author)
  • René Günther - , Department of Neurology, TUD Dresden University of Technology (Author)
  • Claudia D Wurster - , Ulm University Medical Center (Author)
  • Christoph Kleinschnitz - , University Hospital Essen (Author)

Abstract

With Nusinersen, a first causative treatment for 5q-associated spinal muscular atrophy (SMA) has been available in Europe since 2017. Real-world data from neuromuscular clinical centers in Germany increasingly show a therapeutic benefit of nusinersen also in adult SMA patients of both sexes: in many cases, relevant improvements in or at least a stabilization of motor functions are achieved, potentially leading to enhanced autonomy in activities of daily life and to improved quality of living. Even in patients with severe spinal deformities, intrathecal application is usually feasible and safe using imaging modalities. Regular systematic evaluation of the motor status with validated instruments is crucial for adequate monitoring of the therapeutic effects. The documentation in SMA registries enables systematic development of a database for further development of this novel treatment paradigm. Relevant aspects of this novel therapeutic principle were discussed at an experts conference in Frankfurt / Main in February 2019.

Translated title of the contribution
Spinal Muscular Atrophy - expert recommendations for the use of nusinersen in adult patients

Details

Original languageGerman
Pages (from-to)703-710
Number of pages8
JournalFortschritte der Neurologie, Psychiatrie : FDN
Volume87
Issue number12
Publication statusPublished - Dec 2019
Peer-reviewedYes

External IDs

Scopus 85076832783

Keywords

Keywords

  • Adult, Female, Germany, Humans, Male, Muscular Atrophy, Spinal/drug therapy, Oligonucleotides/therapeutic use