Author(s):
1. Bo Chen:
Zhihui Medicine and Technology College, Dazhou Vocational and Technical College,Dazhou,China
2. Yuanlin Gong:
Academic Affairs office, Dazhou Vocational and Technical College,Dazhou,China
3. Tengteng Zhou:
Zhihui Medicine and Technology College, Dazhou Vocational and Technical College,Dazhou,China
Abstract:
Spinal muscular atrophy (SMA) is a prevalent paediatric neuromuscular disorder characterised by muscle weakness and atrophy resulting from degeneration of spinal cord anterior horn ± motor neurons. Gene therapy formulations exhibit varying bene ts and limitations, driving the need for patient-friendly treatment options tailored to speci c populations. The objective of this meta-analysis was to assess the e ectiveness of gene therapy for motor function in children with SMA. The analysis encompassed a total of 719 participants from six randomised controlled trials (RCTs) conducted between 2017 and 2023. Among the studies, one demonstrated a signi cant and large standardised e ect size (Cohen'sd) favouring nusinersen in terms oHfammersmith Functional Motor Scale Expanded (HFMSE) (d = 0.97) and revised upper limb module (RULM) (d = 0.96). Additionally, another study showed a moderate standardised e ect size (Cohen's d) in favour of nusinersen concerning Hammersmith Infant Neurological Examination-Section 2 (HINE-2) (d = 0.48). However, it is important to note that further research with a longer duration of observation is required to strengthen the evidence.
Page(s):
948-955
Published:
Journal: Journal of College of Physicians and Surgeons--Pakistan : JCPSP, Volume: 34, Issue: 8, Year: 2024
Keywords:
Spinal muscular atrophy
,
Motor function
,
Cohens d
,
Nusinersen
,
Risdiplam
References:
References are not available for this document.
Citations
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