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SMA Therapeutics.pdf

4acd6a_b94333b61aa3469d9975be9998bf32c2.pdf

🔗 Source: sumnerlab.com

📊 Size: 544 KB

👤 Author: Bhavya Ravi, Michelle Harran Chan-Cortés, Charlotte J. Sumner

⬇️ Downloads: 78

Summary

Spinal muscular atrophy (SMA) has seen significant progress with three FDA-approved treatments: nusinersen, onasemnogene abeparvovec, and risdiplam, which target the survival motor neuron (SMN) gene using antisense oligonucleotides, gene replacement therapy, and small molecules. Despite this, clinical outcomes are variable, and optimization requires understanding drug pharmacokinetics, target engagement, and potential toxicities. SMA's unique genetic cascade has led to distinct therapeutic strategies, including modifying SMN2 pre-mRNA splicing and SMN1 gene replacement. Gene-directed therapeutics, such as antisense oligonucleotides, have shown clinical success in SMA, with nusinersen and risdiplam promoting exon 7 inclusion in SMN2 transcripts, and onasemnogene abeparvovec replacing the SMN1 gene.

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Description

Spinal muscular atrophy (SMA) has seen significant progress with three FDA-approved treatments: nusinersen, onasemnogene abeparvovec, and risdiplam, which...

Technical Information

File Format: PDF
File Size: 544 KB
Pages: 17
Language: EN
Author: Bhavya Ravi, Michelle Harran Chan-Cortés, Charlotte J. Sumner
Total Downloads: 78
Last Updated: 1 week ago

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