Clinical and Functional Characteristics of a New Phenotype of SMA Type I among a National Sample of Spanish Children: A Cross-Sectional Study
Spinal Muscular Atrophy (SMA) type I has classically presented extremely severe clinical features. New pharmacological treatments have led to a new phenotype of SMA. The aim of this study was to describe the current health and functional status of children with SMA. A cross-sectional study was condu...
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Main Authors: | Beatriz de- (Author), Javier Güeita-Rodríguez (Author), Domingo Palacios-Ceña (Author), Ángel Luis Rodríguez-Fernández (Author) |
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Format: | Book |
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MDPI AG,
2023-05-01T00:00:00Z.
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