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鈥�
- Investigators
- Matthew R Burns, Sub Subramony
- Ages
- 6 Years - N/A
- 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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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鈥�
- 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鈥�
- Ages
- 5 Years - N/A
- 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
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IMPALA-2 160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim or placebo for 48 weeks. Subjects completing the 48 week placebo-controlled period will receive鈥�
- Investigator
- Ali Ataya
- Ages
- 18 Years - N/A
- Sexes
- All
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ROR-PH-303 (APD811-303) Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension (OLE) study for participants with WHO Group 1 PAH who have participated in another Phase 2 or Phase 3 study of ralinepag.
- Investigator
- Ali Ataya
- Ages
- 18 Years - N/A
- Sexes
- All