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Autor:
Kristine S Ojala, Cassandra J Kaufhold, Mykenzie R Davey, Donggyun Yang, Mary Liang, Peter Wipf, Yomna Badawi, Stephen D Meriney
Publikováno v:
Human Molecular Genetics. 32:1901-1911
Spinal muscular atrophy (SMA) is a monogenic disease that clinically manifests as severe muscle weakness owing to neurotransmission defects and motoneuron degeneration. Individuals affected by SMA experience neuromuscular weakness that impacts functi