Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies
Abstract Friedreich Ataxia (FA) is an incurable neurodegenerative disease with systemic consequences affecting vital organs including those of the central and peripheral nervous systems. This article will use FA as an example to explore some of the practical and ethical issues emerging in precision...
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| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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BMC
2024-11-01
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| Series: | International Journal for Equity in Health |
| Online Access: | https://doi.org/10.1186/s12939-024-02318-w |
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| _version_ | 1850196414438572032 |
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| author | Faith A. A. Kwa Evie Kendal |
| author_facet | Faith A. A. Kwa Evie Kendal |
| author_sort | Faith A. A. Kwa |
| collection | DOAJ |
| description | Abstract Friedreich Ataxia (FA) is an incurable neurodegenerative disease with systemic consequences affecting vital organs including those of the central and peripheral nervous systems. This article will use FA as an example to explore some of the practical and ethical issues emerging in precision medicine for rare diseases. It will first describe the existing management strategies available for FA patients, before considering the potential impact of gene therapy trials on the prevention and treatment of disease symptoms. Finally, ethical considerations will be discussed, including equity of access and managing resource allocation dilemmas; balancing benefits, burdens and harms; and gaining informed consent for novel treatments. |
| format | Article |
| id | doaj-art-34d31240c4cd45ce85cce3c869c43e0a |
| institution | OA Journals |
| issn | 1475-9276 |
| language | English |
| publishDate | 2024-11-01 |
| publisher | BMC |
| record_format | Article |
| series | International Journal for Equity in Health |
| spelling | doaj-art-34d31240c4cd45ce85cce3c869c43e0a2025-08-20T02:13:27ZengBMCInternational Journal for Equity in Health1475-92762024-11-012311710.1186/s12939-024-02318-wPrecision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapiesFaith A. A. Kwa0Evie Kendal1School of Health Sciences, Swinburne University of TechnologySchool of Health Sciences, Swinburne University of TechnologyAbstract Friedreich Ataxia (FA) is an incurable neurodegenerative disease with systemic consequences affecting vital organs including those of the central and peripheral nervous systems. This article will use FA as an example to explore some of the practical and ethical issues emerging in precision medicine for rare diseases. It will first describe the existing management strategies available for FA patients, before considering the potential impact of gene therapy trials on the prevention and treatment of disease symptoms. Finally, ethical considerations will be discussed, including equity of access and managing resource allocation dilemmas; balancing benefits, burdens and harms; and gaining informed consent for novel treatments.https://doi.org/10.1186/s12939-024-02318-w |
| spellingShingle | Faith A. A. Kwa Evie Kendal Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies International Journal for Equity in Health |
| title | Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies |
| title_full | Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies |
| title_fullStr | Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies |
| title_full_unstemmed | Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies |
| title_short | Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies |
| title_sort | precision medicine and friedreich ataxia promoting equity beneficence and informed consent for novel gene therapies |
| url | https://doi.org/10.1186/s12939-024-02318-w |
| work_keys_str_mv | AT faithaakwa precisionmedicineandfriedreichataxiapromotingequitybeneficenceandinformedconsentfornovelgenetherapies AT eviekendal precisionmedicineandfriedreichataxiapromotingequitybeneficenceandinformedconsentfornovelgenetherapies |