Rare SMA Patients: A Comprehensive Look at Clinical Features, Genetic Profiles and Therapeutic Approaches
Kristina Mikhalchuk, Svetlana Artemieva, Viktoria Zabnenkova, Maria Akhkiamova et autres
Spinal muscular atrophy 5q (5q SMA) is one of the most prevalent autosomal recessive disorders globally. The underlying cause of 5q SMA is attributed to variants in SMN1. To date, there are no reported cases of gene-based therapy in rare patients with …
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International Journal of Molecular Sciences