Key result
Initial SMA disease-modifying treatments retain ~64% of patients, with switches driven by new drug approvals.
Why the study?
Real-world treatments for 5q-spinal muscular atrophy have evolved rapidly following the sequential approval of three disease-modifying therapies, prompting the need to accurately map treatment sequence and timing.
What are the real-world treatment patterns and factors influencing treatment switches in patients with spinal muscular atrophy?
Population
2,140 patients with SMA across 84 centers in Germany, Austria, and Switzerland
Comparison
Patients remaining on first DMT vs those who switched DMT
Design
Multicenter registry study
Authors
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In a large real-world registry of SMA patients, most remained on their initial disease-modifying therapy, with treatment switches primarily driven by new drug approvals rather than changes in motor function.
What are the real-world treatment patterns and factors influencing treatment switches in patients with spinal muscular atrophy?
In a large real-world registry of SMA patients, most remained on their initial disease-modifying therapy, with treatment switches primarily driven by new drug approvals rather than changes in motor function.
Voigt-Müller et al. (2025) studied this question. In a cohort of 2,140 SMA patients, 63.8% stayed on the first disease-modifying treatment, with most switches occurring after new treatment approvals.
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