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Researchers from Children's Hospital Philadelphia Report Findings in Friedreich Ataxia (Evaluating Mfars In Pediatric Friedreich's Ataxia: Insights From the Fachild Study).
- Source :
- Medical Letter on the CDC & FDA; 4/26/2024, p1999-1999, 1p
- Publication Year :
- 2024
-
Abstract
- A recent report discusses research on Friedreich Ataxia, a rare genetic disorder that causes progressive coordination loss and other symptoms. The study, conducted by researchers from Children's Hospital Philadelphia, aimed to expand knowledge about the disease's natural history and clinical outcome assessments for pediatric patients. The study enrolled 108 individuals aged 7-18 years with a confirmed diagnosis of Friedreich Ataxia. The results confirmed the usefulness of the mFARS score in children but also highlighted issues with the upper limb subscore. The study suggests that the FARS E (Upright Stability) score may be a preferred outcome measure for this patient population. [Extracted from the article]
Details
- Language :
- English
- ISSN :
- 15324648
- Database :
- Supplemental Index
- Journal :
- Medical Letter on the CDC & FDA
- Publication Type :
- Periodical
- Accession number :
- 176679962