Article

Practical Recommendations for the Selection of Patients for Individualized…

Human Mutation · 2023 · Bianca Zardetto, author Marlen C. Lauffer, author Willeke van Roon-Mom, author Annemieke Aartsma-Rus, author on behalf of the N = 1 Collaborative, author Brage Andresen, Academic Editor

Key facts

Title Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments
First author Bianca Zardetto, author Marlen C. Lauffer, author Willeke van Roon-Mom, author Annemieke Aartsma-Rus, author on behalf of the N = 1 Collaborative, author Brage Andresen, Academic Editor
Journal Human Mutation
Published May 30, 2023
DOI 10.1155/2024/9920230
Devices mentioned 017411

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  • 1–9 FDA submissions mentioned
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