Longitudinal analysis shows GAA1 length and baseline clinical status as robust predictors of progression in Friedreich ataxia
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Friedreich's ataxia (FRDA) is the most common early onset hereditary ataxia, caused by GAA repeat expansions in the FXN gene. The length of the shorter allele (GAA1) and age at onset are established determinants of disease severity, though additional biomarke... ...
