ArticleJournal of neuromuscular diseases2026
Clinical and functional outcome measures in LAMA2-related muscular dystrophy and SELENON-related myopathy; a 1.5-year natural history study.
Article in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
BackgroundLAMA2-related muscular dystrophy (LAMA2-MD) and SELENON-related myopathy (SELENON-RM) are rare congenital muscle diseases characterized by slowly progressive proximal muscle weakness, spinal rigidity and respiratory insufficiency. The LAST STRONG study is a natural history study to identify suitable outcome measures and reach trial readiness for LAMA2-MD and SELENON-RM.MethodsPatients had four visits over 1.5-years. Assessments included neurological examination, hand-held dynamometry (HHD), functional assessments (Motor Function Measurements(MFM)-20/32, graded and timed function tests), accelerometry, and questionnaires on quality of life, activities and participation, pain, and fatigue.ResultsA total of 27 LAMA2-MD (21 years, range 3-50; 9 males) and 11 SELENON-RM (20 years, range 3-42; 8 males) patients were included. In LAMA2-MD, mean HHD scores for neck extensors, biceps brachii, quadriceps, foot plantar flexors and handgrip strength increased (all p < 0.02). In SELENON-RM, biceps brachii and foot plantar flexors increased (p < 0.01). In LAMA2-MD, functional assessments did not change significantly. In SELENON-RM, MFM-20/32 total score and domain 1 decreased (p < 0.05), 6-Minute Walk Test (6MWT) decreased (p < 0.01) and 10-Meter Walk Test (10MWT) increased (p < 0.01). Accelerometry showed a significant change in moderate activity in LAMA2-MD (p <0.01), no changes were observed in SELENON-RM.DiscussionOver 1.5 years, LAMA2-MD remained mostly stable, while SELENON-RM showed minimal functional decline on select outcome measures. Both cohorts showed HHD increases, likely reflecting age-related development. Most measures did not capture disease progression, however MFM-20/32, 6MWT and 10MWT showed small significant changes in SELENON-RM, suggesting potential trial endpoints.
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