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Currently approved exon-skipping therapies target exons 44, 45, 51, and 53. Since your child has a 3-17 exon duplication, the currently available exon-skipping medications would not be effective treatments. It's important to discuss specific treatment options with your child's healthcare team since they will have detailed Show Full Answer

Currently approved exon-skipping therapies target exons 44, 45, 51, and 53. Since your child has a 3-17 exon duplication, the currently available exon-skipping medications would not be effective treatments. It's important to discuss specific treatment options with your child's healthcare team since they will have detailed knowledge of your child's specific genetic mutation. While exon-skipping therapy may not be an option right now, researchers are actively working on developing new versions of these treatments that target other exons. Your healthcare provider can discuss whether participating in clinical trials might be appropriate for your child, as well as other current treatment approaches to help manage symptoms and slow disease progression.

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