Key result
Disease-modifying therapies transform pediatric SMA outcomes but yield muted responses in adolescents and adults.
Why the study?
Despite therapeutic advances, ongoing challenges, plateau effects, and unmet needs remain for individuals with spinal muscular atrophy, especially adolescents and adults.
While disease-modifying therapies have revolutionized pediatric SMA care, older patients experience plateau effects and new phenotypic challenges requiring innovative solutions.
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May temper DMT expectations in adolescent/adult SMA; leaves open need for age-specific therapies.
René et al. (2026) conducted a review in Spinal muscular atrophy. Disease-modifying therapies was evaluated. Disease-modifying therapies have dramatically changed the clinical course for pediatric spinal muscular atrophy, though adolescent and adult individuals frequently experience more muted responses.
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