ArticleNeurogenetics2026
Retrospective analysis of spinal muscular atrophy in tunisian population: phenotypic-genotypic associations and considerations for therapeutic advancement.
Article in Neurogenetics, 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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10 authors.
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Abstract
This study retrospectively analyzed 97 Tunisian Spinal Muscular Atrophy (SMA) patients (2008-2024), characterizing their phenotypes, diagnosis age, and consanguinity alongside copy number variations of SMN1, SMN2, NAIP, p44, and H4F5. SMA-I was the most prevalent phenotype (61.86%). We notice a significant male predominance with significant late age of diagnosis. Consanguinity was remarkably high (73.53%) and strongly associated with severe form SMA-I. Genetic analysis confirmed SMN2 and NAIP as key modifiers, where lower copy numbers correlated with increased severity and respiratory complications. With 98% of patients carrying ≥ 2 SMN2 copies, most are eligible for splicing-modifier therapies. Additionally, the prevalence of early-onset SMA-I underscores a promising perspective for gene replacement therapy before irreversible motor neuron loss occurs. SMN2 and NAIP are vital prognostic markers in Tunisia. High consanguinity and gender-based diagnostic delays necessitate urgent newborn screening and early genetic intervention to optimize modern therapy efficacy.
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