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| Home > Publications Database > The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap. |
| Home > Publications Database > The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap. |
| Journal Article (Review Article) | DZNE-2025-00045 |
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2025
Nature Publ. Group
London
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Please use a persistent id in citations: doi:10.1038/s41573-024-01059-3
Abstract: In recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases. In this Review, we outline the concept of N-of-1 individualized therapies, focusing on genetic therapies, and illustrate advances and challenges in the field using cases for which therapies have been successfully developed. We discuss why the traditional drug development and reimbursement pathway is not fit for purpose in this field, and outline the pragmatic, regulatory and ethical challenges this poses for future access to N-of-1 therapies. Finally, we provide a roadmap for N-of-1 individualized therapy development.
Keyword(s): Single-Case Studies as Topic (MeSH) ; Humans (MeSH) ; Drug Development: methods (MeSH) ; Precision Medicine: methods (MeSH) ; Genetic Therapy: methods (MeSH) ; Genetic Therapy: trends (MeSH) ; Rare Diseases: drug therapy (MeSH) ; Rare Diseases: genetics (MeSH)
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