Article

Treatment of 5q-spinal muscular atrophy with nusinersen in Southern China: A single center retrospective study

2023-03-08

Abstract excerpt

Nusinersen is the first approved disease-modifing therapy for all 5q-spinal muscular atrophy patients regardless of age or disease severity. In 2019, nusinersen treatment was approved by China Food and Drug Administration. Here, we first present the results of nusinersen treatment in Southern China. We analyzed clinical and laboratory data from 45 5q-spinal muscular atrophy patients between December 2019 and May 2...

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Literature Corpus work: 6ef69243-c983-50f6-834d-90c5e9e1c772
DOI: 10.21203/rs.3.rs-2566569/v1

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