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  • Publication : 28 11 2019
  • Catégorie :La recherche médicale et génétique

Nusinersen for older patients with SMA: a real-world clinical setting experience.    

Abstract
INTRODUCTION: Clinical trials data concerning use of nusinersen in older spinal muscular atrophy (SMA) patients is lacking. We describe our center's experience in using intrathecal nusinersen for older patients in the clinical setting.
METHODS: Retrospective study.
RESULTS: 12 patients (12 - 52?years old) were treated with nusinersen. Mean follow-up duration was 17.4 months (4-26). All patients had scoliosis; 10 had spinal fusion/instrumentation. All procedures (30 cervical and 57 lumbar punctures) were technically successful. The only side effects were post-procedural headache (9%) and site pain (5.7%). Functional assessments showed stability in 6/9 patients and improvement in 3/9 patients. Subjective improvements in endurance, hand strength, and bulbar functioning critical for activities of daily living were reported in 8/12 patients. None of the patients has discontinued treatment so far.
DISCUSSION: Intrathecal nusinersen can be safely delivered in older SMA patients. Available functional outcome measures are not adequate to capture meaningful subjective improvements. This article is protected by copyright. All rights reserved.

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