Clinical trials

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RAN proteins in Ataxia -
Respiratory training in Friedreich's ataxia - Investigator
- Carmen Leon Astudillo
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Stimulation-induced ataxia in essential tremor - Investigator
- Adolfo Ramirez-Zamora
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Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as…
- Investigator
- Sub Subramony
- Ages
- 6 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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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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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
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United therapeutics 301 Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO)…
- Investigator
- Ali Ataya
- Ages
- 18 Years - N/A
- Sexes
- All
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Evaluate the Efficacy, Safety and Pharmacokinetics of Treprostinil Palmitil Inhalation Powder The main objective of the study is to assess the effect of treprostinil palmitil inhalation powder (TPIP) compared with placebo on pulmonary vascular resistance.
- Investigator
- Ali Ataya
- Ages
- 18 Years - 75 Years
- Sexes
- All
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Sapphire This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.
- Investigator
- Ali Ataya
- Ages
- 18 Years - 99 Years
- Sexes
- All