Article

Cerebellar pathology contributes to neurodevelopmental deficits in spinal muscular atrophy

2025-06-11

Abstract excerpt

<title>Abstract</title> <p> Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by ubiquitous SMN deficiency and loss of motor neurons. The persistence of motor and communication impairments, together with emerging cognitive and social deficits in severe Type I SMA patients treated early with SMN-restoring therapies, suggests a broader dysfunction involving neural circuits of the brain. To exp...

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Literature Corpus work: 20fef616-3957-5ec3-a454-81524a72a026
DOI: 10.21203/rs.3.rs-6819992/v1

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