Key result
Among 107 German patients with spinal muscular atrophy, 78% received disease-modifying therapies, but significant gaps remained in rehabilitation, respiratory care, and adult employment.
Why the study?
Little is known about the uptake and impact of novel transformative and potentially curative therapies for spinal muscular atrophy in real-world clinical practice.
Population
107 patients with genetically confirmed SMA (24 children and 83 adults) in Germany
Design
Registry-based cross-sectional observational study
Authors
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Care gaps in SMA rehabilitation and support persist despite DMT access; leaves open optimal multidisciplinary models for adults.
Cross-Sectional (n=107)
The natural history of spinal muscular atrophy has improved in Germany following the introduction of novel therapies, though significant gaps remain in rehabilitation, respiratory care, and treatment access.
Leibrock et al. (2023) conducted a cross-sectional in Spinal muscular atrophy (SMA) (n=107). Spinal muscular atrophy was evaluated on Current motor function, medical care, and socioeconomic status. Among 107 German patients with spinal muscular atrophy, 78% received disease-modifying therapies, but significant gaps remained in rehabilitation, respiratory care, and adult employment.
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