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...
保存先:
| 主要な著者: | , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Wiley
2024-05-01
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| シリーズ: | Annals of Clinical and Translational Neurology |
| オンライン･アクセス: | https://doi.org/10.1002/acn3.52057 |
| タグ: |
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