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Clinical trials

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  • Interposed Nucleus aDBS for Ataxia

    This is a single-center, open-label study designed to evaluate the feasibility, safety, and preliminary efficacy of cerebellar adaptive deep brain stimulation (aDBS) in adults with spinocerebellar ataxia type 6 (SCA6). A total of 5 participants will…

    Ages
    21 Years - 89 Years
    Sexes
    All
  • 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…

    Investigators
    Sub Subramony, Matthew R Burns
    Ages
    6 Years - N/A
    Sexes
    All
  • 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
  • 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
  • GB002-3102

    This open-label extension study will evaluate the long-term safety, tolerability and efficacy of orally inhaled seralutinib in subjects who have completed a previous seralutinib study

    Investigator
    Ali Ataya
    Ages
    18 Years - 75 Years
    Sexes
    All