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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
  • 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
  • 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
  • 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
  • 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
  • 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