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IFN-γ for Friedreich ataxia: present evidence

    McKenzie Wells

    Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA

    Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA

    ,
    Lauren Seyer

    Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA

    Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA

    ,
    Kimberly Schadt

    Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA

    Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA

    &
    David R Lynch

    *Author for correspondence:

    E-mail Address: lynchd@mail.med.upenn.edu

    Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA

    Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA

    Published Online:https://doi.org/10.2217/nmt.15.52

    IFN-γ-1b is currently US FDA approved as an orphan drug for the treatment of chronic granulomatous disease and severe malignant osteopetrosis. It is administered via subcutaneous injection and is a potential therapy for Friedreich ataxia (FRDA), a rare degenerative neurological condition. Ongoing Phase II and III trials in both adults and children with FRDA were preceded by a small Phase I, open-label trial in children that showed that IFN-γ-1b was reasonably well-tolerated and improved overall neurological function as measured by the Friedreich Ataxia Rating Scale after 12 weeks of treatment, though the primary outcome measure of frataxin level showed no improvement. Although there is an established dose of IFN-γ-1b prescribed for the current indications, the efficacy and tolerability of these dose levels in the FRDA population remains the subject of ongoing investigation.

    Papers of special note have been highlighted as: • of interest; •• of considerable interest

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