Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec-Preliminary Results

Spinal muscular atrophy (SMA) is a rare genetic disorder, with the most common form being 5q SMA. Survival of children with severe SMA is poor, yet major advances have been made in recent years in pharmaceutical treatment, such as gene-therapy, which has improved patient survival. Therefore, clinica...

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Main Authors: Venla Soini (Author), Gudrun Schreiber (Author), Bernd Wilken (Author), Anna Kathrin Hell (Author)
Format: Book
Published: MDPI AG, 2023-06-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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