Article

Effect of nusinersen after three years of treatment in 61 young children with SMA type 1 or 2: a French real-life observational study

2022-08-23

Abstract excerpt

<title>Abstract</title> <p>Background Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder due to an autosomal recessive mutation in the survival motor neuron 1 gene (<italic>SMN1</italic>) causing degeneration of the anterior horn cells of the spinal cord and resulting in muscle atrophy. The aim of this paper is to report a 36-month follow-up of children with SMA treated with nusinersen before...

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Literature Corpus work: bb56672e-1e54-572b-b1cd-258e65a9df5d
DOI: 10.21203/rs.3.rs-1932236/v1

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