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Impaired Neurodevelopment in Children with 5q-SMA - 2 Years After Newborn Screening.

J Neuromuscul Dis · 2024 · PMC10789341 · PMID 37927272

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a positive trendno p-value reported
In symptomatic patients with SMA type 1 treated with disease-modifying therapies, global developmental delay and defects in gross motor functions have been found in the majority, whereas scores obtained on scales to assess learning and language skills suggest a positive trend in the development of general neurocognitive skills [ 31 ].

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