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Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophy

18 Apr 2025

Abstract excerpt

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder characterized by progressive muscular weakness due to the loss of lower motor neurons. The most severe and common form, SMA type 1 (Werdnig-Hoffmann disease), is a devastating childhood condition. Exegenesis Bio is developing EXG001-307, a recombinant adeno-associated virus (rAAV) gene therapy, for SMA type 1 patients lacking...

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PMID: 40458203
DOI: 10.1016/j.omtm.2025.101475

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