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Friedreich’s ataxia (fa) is a rare genetic childhood condition with a variety of symptoms, including trouble with balance, coordination, and a progressive loss of mobility that ultimately leads to wheelchair dependency. A total of 42 individuals (29 adults and 13 children) with frda were recruited In this article we review those clinical trials with a focus on the instruments used as endpoints to assess clinical progression, and discuss the potential benefits of integrating additional measures, including assessments from overlooked domains.
Given the abundance of neurological natural history data, fara believes these data a e informative for designing trials, providing outcome assessment e.g., employing bayesian approaches to reduce size of placebo group in trial design We performed a prospective observational cohort study to evaluate the efficacy of inpatient rehabilitation (ir) for people with frda Can fda comment on the proposed utility of natural history data?
In light of these developments, we review milestones in frda translational and clinical research over the past 10 years, as well as the various therapeutic strategies currently in the.
The consortium collaborates with pharmaceutical companies, government agencies, other research centers, and the patient community to facilitate clinical research and trials needed to identify new therapies.
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