Progress in the application of assessment scales associated with activities of daily living in the disease-modifying treatments of spinal muscular atrophy
10.3760/cma.j.issn.1673-4408.2023.06.003
- VernacularTitle:脊髓性肌萎缩症疾病修正治疗中患者日常生活活动评估量表的应用进展
- Author:
Yingshuang PENG
1
;
Siqi HONG
Author Information
1. 重庆医科大学附属儿童医院神经内科 400014
- Keywords:
Spinal muscular atrophy;
Disease-modifying treatments;
Activities of daily living;
Quality of life
- From:
International Journal of Pediatrics
2023;50(6):370-373
- CountryChina
- Language:Chinese
-
Abstract:
Spinal muscular atrophy(SMA) is a serious neuromuscular degenerative disease that severely impairs the quality of life for patients and entire families.The emergence of disease-modifying treatments such as nusinersen and risdiplam has gradually changed the natural course of SMA patients.It is particularly important to include the activities of daily living(ADL) ability reported by patients or caregivers in the comprehensive assessment of SMA patients.There are many ADL-related assessment tools, and studies have found that disease-modifying treatments can somewhat improve the ADL ability of SMA children.This article reviews the progress on the effect of disease-modifying treatments on ADL in SMA patients, which can provide a reference for exploring more comprehensive and effective assessment tools and treatment decision-making in subsequent clinical practice.
