SMA – OUTCOME MEASURES AND REGISTRIES
نویسندگان
چکیده
Nusinersen was approved as the first medical treatment for all patients with spinal muscular atrophy (SMA). A recent study in a large cohort of adult treated nusinersen demonstrated significant improvements Hammersmith functional motor scale expanded (HFMSE). However, clinically meaningful were seen only 40% at 14 months despite positive patients’ experience. The main issue open label remains risk placebo effect, which could be largely compensated by use continuous and passive measures. We have longitudinal natural history that drop-in activity measured over period 1 year SMA using magneto-inertial technology. aimed to assess efficacy adults standard outcomes measure issued from wearable device. In order achieve this, 13 (6 ambulant, 7 non-ambulant) type 2 3 aged 23 59 years, assessed before every 4 after initiation. Evaluations included HFMSE, function measure, revised upper limb module, dynamometric measures, 6-minute walk, 4-stair climb, rise floor, 10-metre walk tests. Around each visit, wore sensor ankle wrist. Baseline HFMSE 30.27± 23.04. scores change ± 3.19. non-ambulant patients, no decline observed different variables computed (power level, vertical component movement), contrasts data drop around 5% observed. ambulant stride length, velocity maximal distance walked remain stable months.
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ژورنال
عنوان ژورنال: Neuromuscular Disorders
سال: 2021
ISSN: ['0960-8966', '1873-2364']
DOI: https://doi.org/10.1016/j.nmd.2021.07.290