Crigler-Najjar syndrome: looking to the future does not make us forget the present
Abstract Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore, there is a risk of overlooking several unresol...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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BMC
2024-03-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-024-03108-x |
| _version_ | 1797266655366086656 |
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| author | Fabiola Di Dato Giuseppe D’Uonno Raffaele Iorio |
| author_facet | Fabiola Di Dato Giuseppe D’Uonno Raffaele Iorio |
| author_sort | Fabiola Di Dato |
| collection | DOAJ |
| description | Abstract Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore, there is a risk of overlooking several unresolved problems still present in current clinical practice. This letter is a call for action on crucial open issues that remain nowadays an unmet need in the management of CNS patients. |
| first_indexed | 2024-04-25T01:04:08Z |
| format | Article |
| id | doaj.art-b42ada63175a4cdca5f8a8efa3ad80cb |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-04-25T01:04:08Z |
| publishDate | 2024-03-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-b42ada63175a4cdca5f8a8efa3ad80cb2024-03-10T12:21:59ZengBMCOrphanet Journal of Rare Diseases1750-11722024-03-011911310.1186/s13023-024-03108-xCrigler-Najjar syndrome: looking to the future does not make us forget the presentFabiola Di Dato0Giuseppe D’Uonno1Raffaele Iorio2Department of Translational Medical Science, Section of Pediatrics, University of Naples Federico IIDepartment of Translational Medical Science, Section of Pediatrics, University of Naples Federico IIDepartment of Translational Medical Science, Section of Pediatrics, University of Naples Federico IIAbstract Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore, there is a risk of overlooking several unresolved problems still present in current clinical practice. This letter is a call for action on crucial open issues that remain nowadays an unmet need in the management of CNS patients.https://doi.org/10.1186/s13023-024-03108-xGene therapyLiver transplantationPhenobarbitalPhototherapy |
| spellingShingle | Fabiola Di Dato Giuseppe D’Uonno Raffaele Iorio Crigler-Najjar syndrome: looking to the future does not make us forget the present Orphanet Journal of Rare Diseases Gene therapy Liver transplantation Phenobarbital Phototherapy |
| title | Crigler-Najjar syndrome: looking to the future does not make us forget the present |
| title_full | Crigler-Najjar syndrome: looking to the future does not make us forget the present |
| title_fullStr | Crigler-Najjar syndrome: looking to the future does not make us forget the present |
| title_full_unstemmed | Crigler-Najjar syndrome: looking to the future does not make us forget the present |
| title_short | Crigler-Najjar syndrome: looking to the future does not make us forget the present |
| title_sort | crigler najjar syndrome looking to the future does not make us forget the present |
| topic | Gene therapy Liver transplantation Phenobarbital Phototherapy |
| url | https://doi.org/10.1186/s13023-024-03108-x |
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