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Transitional Life Events in Friedreich Ataxia: Differential Age at Onset PerspectivesNARA Subscribed
Friedreich’s ataxia (FA) is a rare, autosomal recessive neurodegenerative disease that primarily affects children and adolescents in their transition to adulthood, which is associated with several adverse life events caused by the disease progression. This article aims to describe life events associated with FA and their impact on patient-reported outcomes in pediatric and adult onsets. A newly adapted Life Events Questionnair...
A Patient-Reported Outcome Measure of Communication Difficulties in Friedreich Ataxia: COMATAX.NARA Subscribed
Background and Objectives: Friedreich ataxia (FA) causes progressive impairment of communication due to gradual deterioration of speech, associated with impaired hearing, socio-cognitive and language skills. There is an urgent need to investigate the impact of this multiparametric alteration on patients’ lives. Therefore, the COMunication and ATAXia measure (COMATAX) was developed and validated in French and German. Methods: I...
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