Advances and limitations for the treatment of spinal muscular atrophy

Advances and limitations for the treatment of spinal muscular atrophy

Abstract Spinal muscular atrophy (5q-SMA; SMA), a genetic neuromuscular condition affecting spinal motor neurons, is caused by defects in both copies of the SMN1 gene that produces survival motor neuron (SMN) protein. The highly homologous SMN2 gene primarily expresses a rapidly degraded isoform of...

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Main Authors: John W. Day (Author), Kelly Howell (Author), Amy Place (Author), Kimberly Long (Author), Jose Rossello (Author), Nathalie Kertesz (Author), George Nomikos (Author)
Format: Book
Published: BMC, 2022-11-01T00:00:00Z.
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3rd Floor Main Library

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