Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD study
Abstract Objectives Friedreich ataxia (FRDA) is a rare genetic disorder caused by mutations in the FXN gene, leading to progressive coordination loss and other symptoms. The recently approved omaveloxolone targets this condition but is limited to patients over 16 years of age, highlighting the need...
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| Glavni autori: | , , , , |
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| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
Wiley
2024-05-01
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| Serija: | Annals of Clinical and Translational Neurology |
| Online pristup: | https://doi.org/10.1002/acn3.52057 |
| Oznake: |
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