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In utero therapy for spinal muscular atrophy: closer to clinical translation

7 Apr 2025

Abstract excerpt

5q-Spinal muscular atrophy (SMA) has been a trailblazer in the development of advanced therapies for inherited diseases. SMA is an autosomal recessive disorder affecting mainly motor neurons in the anterior horn of the spinal cord and brainstem motor nucle but currently considered a systemic disease. Advances in understanding the genetics of SMA led to the development of disease-modifying therapies, either...

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PMID: 40193572
DOI: 10.1093/brain/awaf123

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