Clinical Trial: Measurement of Frataxin mRNA in Biofluids
Study Status: COMPLETED
Recruit Status: COMPLETED
Condition: Friedreich Ataxia
Study Type: OBSERVATIONAL
Official Title: Measurement of Frataxin mRNA in NHP and Human Biofluids in Patients With Friedreich's Ataxia
Brief Summary:
The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA).The gene mutation in FRDA leads to low levels of the mRNA and then low levels of the protein frataxin that leads to the disease.Treatments being developed for FRDA have the ability increase these levels including in brain where it is needed.Currently, there is no accepted way to measure frataxin protein or the…
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