Article

Precisely Correcting SMN Deficiency Pathways in Spinal Muscular Atrophy Using an Off-Amyloid Stabilizer

2024-07-11

Abstract excerpt

<title>Abstract</title> <p>While therapies like Spinraza, Zolgensma, and Evrysdi focus on increasing SMN1 protein levels to treat spinal muscular atrophy (SMA), their effectiveness is limited and highly dependent on early intervention. This indicates that merely augmenting SMN levels may not adequately reverse the permanent pathological effects stemming from SMN1 deficiency. Our research indicates that SMA is a p...

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Literature Corpus work: 21cbd671-14df-5d10-a1e4-f194409dd342
DOI: 10.21203/rs.3.rs-4586057/v1

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