- Investigator
- Carmen Leon Astudillo
Clinical trials
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Respiratory training in Friedreich's ataxia -
RAN proteins in Ataxia -
JIVE (LARIMAR) To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).
- Investigator
- Sub Subramony
- Ages
- 18 Years - N/A
- Sexes
- All
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Frataxin 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…
- Investigator
- Sub Subramony
- Ages
- 18 Years - 65 Years
- Sexes
- All
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TRACK-FA This is a natural history study prospectively investigating neuroimaging markers of disease progression in children and adults with Friedreich ataxia (FA). There will be three assessment periods (baseline, 12 and 24 months). The study will include…
- Ages
- 5 Years - N/A
- Sexes
- All