Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy.

Alma OsmanovicGresa RanxhaMareike KumpeClaudia D WursterBenjamin StolteIsabell CordtsRené GüntherMaren FreigangLars H MüschenCamilla BinzAndreas HermannMarcus DeschauerPaul LingorAlbert C LudolphTim HagenackerOlivia Schreiber-KatzSusanne Petri

Published in: Therapeutic advances in neurological disorders (2021)

Most patients were satisfied with nusinersen treatment effectiveness. Less severely affected patients indicated higher satisfaction. The TSQM-1.4© helped to identify therapy non-responders, who mainly addressed dissatisfaction with effectiveness and convenience. We suggest introducing the TSQM-1.4© as an additional PRO in SMA into clinical practice.

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
randomized controlled trial
systematic review
peritoneal dialysis
prognostic factors
stem cells
bone marrow
replacement therapy
cell therapy