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Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders

4 Jan 2022

Abstract excerpt

BACKGROUND: Major efforts have been made in the last decade to develop and improve therapies for proximal spinal muscular atrophy (SMA). The introduction of Nusinersen/Spinraza™ as an antisense oligonucleotide therapy, Onasemnogene abeparvovec/Zolgensma™ as an AAV9-based gene therapy and Risdiplam/Evrysdi™ as a small molecule modifier of pre-mRNA splicing have set new standards for interference with...

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PMID: 34983696
DOI: 10.1186/s42466-021-00162-9

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