Article

Combining SMN2 splicing modifiers with HDAC6 inhibition greatly improves muscle function and survival in Spinal Muscular Atrophy

2025-01-20

Abstract excerpt

Spinal muscular atrophy (SMA) is a rare, progressive and severe neuromuscular disease. It is mostly caused by mutations in the SMN gene, which lead to the death of spinal cord motor neurons. In the absence of treatment, more than half of affected children die before the age of two. Recently, groundbreaking gene therapies were developed, allowing children to survive. However, a new clinical presentation of the dise...

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Literature Corpus work: ae20c8b6-975d-537e-8aec-b1e1dbb28c4d
DOI: 10.1101/2025.01.15.633267

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