Spinale Muskelatrophie – Expertenempfehlungen zur Behandlung von erwachsenen Patienten mit Nusinersen

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Tim Hagenacker - , Universitätsklinikum Essen (Autor:in)
  • Andreas Hermann - , Universitätsmedizin Rostock (Autor:in)
  • Christoph Kamm - , Universitätsmedizin Rostock (Autor:in)
  • Maggie C Walter - , Klinikum der Ludwig-Maximilians-Universität (LMU) München (Autor:in)
  • Markus Weiler - , Universitätsklinikum Heidelberg (Autor:in)
  • René Günther - , Klinik und Poliklinik für Neurologie, Technische Universität Dresden (Autor:in)
  • Claudia D Wurster - , Universitätsklinikum Ulm (Autor:in)
  • Christoph Kleinschnitz - , Universitätsklinikum Essen (Autor:in)

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.

Details

OriginalspracheDeutsch
Seiten (von - bis)703-710
Seitenumfang8
FachzeitschriftFortschritte der Neurologie, Psychiatrie : FDN
Jahrgang87
Ausgabenummer12
PublikationsstatusVeröffentlicht - Dez. 2019
Peer-Review-StatusJa

Externe IDs

Scopus 85076832783

Schlagworte

Schlagwörter

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