ArticleZhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics2021
Natural history of spinal muscular atrophy in children: an analysis of 117 cases.
Article in Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed, 2 citations in OpenAlex.
- A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results.Advances in therapy · 2024Article
- Motor and neurocognitive profiles of children with symptomatic spinal muscular atrophy type 1 with two copies ofFrontiers in neurology · 2024Article
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Authors and funding
5 authors at 1 institution in 1 country.
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Abstract
objectivesTo study the natural history of spinal muscular atrophy (SMA) in Chongqing and surrounding areas, China, and to provide a clinical basis for comprehensive management and gene modification therapy for SMA.
methodsA retrospective analysis was performed on the medical data and survival status of 117 children with SMA.
resultsOf the 117 children, 62 (53.0%) had type 1 SMA, 45 (38.5%) had type 2 SMA, and 10 (8.5%) had type 3 SMA, with a median age of onset of 2 months, 10 months, and 15 months, respectively. Compared with the children with type 2 SMA or type 3 SMA, the children with type 1 SMA had significantly shorter time to onset, consultation, and confirmed diagnosis (
conclusionsThere are differences in clinical manifestations and survival rates among children with different types of SMA. The children with type 1 SMA have a low survival rate, and those with type 2 SMA may have non-linear regression of motor ability. Early identification and management of SMA should be performed in clinical practice.
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