Neurocrine Biosciences
This is a first-in-human study to evaluate the safety, tolerability, and pharmacodynamics of NBIB-0000223 (hereafter referred to as NBIB-223) in participants with Friedreich's Ataxia.
Key Inclusion Criteria: * Has both a clinical and genetic diagnosis of Friedreich's Ataxia, with diagnosis occurring at least 1 year prior to Screening and when the participant was age ≤16 years. * Documented prior genetic diagnosis of Friedreich's Ataxia with a pathogenic GAA trinucleotide repeat expansion in intron 1 of both frataxin alleles, based on analysis by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory. * Clinical evidence of symptomatic neurologic disease consistent with Friedreich's Ataxia. * Have an Modified Friedreich Ataxia Rating Scale (mFARS) o…
Intravenous (IV) infusion
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