Key result
Among Australians living with spinal muscular atrophy, 81% utilized SMN-augmenting therapies, with 47.6% of treated individuals reporting perceived improvements on the Patient/Parent Global Impression of Improvement scale.
Why the study?
New diagnosis and treatment paradigms have altered the trajectory of SMA, necessitating real-world registry data to evaluate the phenotypic and genotypic landscape, treatment patterns, and health impact in Australia.
Population
195 individuals with SMA enrolled in the Australian Neuromuscular Disease Registry
Design
Registry-based cross-sectional study
Authors
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SMA mortality and multidisciplinary care gaps persist despite therapies; registry data leaves open how best to address evolving needs in treated children and adults.
Cross-Sectional (n=195)
Yes
p-value: p=<0.0001
Despite therapeutic advances, there remains a significant unmet need for multidisciplinary care in both adults and treated children with SMA in Australia.
Balaji et al. (2024) conducted a cross-sectional in Spinal muscular atrophy (n=195). SMN-augmenting therapies vs. No therapy was evaluated on Patient/Parent Global Impression of Improvement (PGI-I) (p=<0.0001). Among Australians living with spinal muscular atrophy, 81% utilized SMN-augmenting therapies, with 47.6% of treated individuals reporting perceived improvements on the Patient/Parent Global Impression of Improvement scale.
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