{
  "abstract": "Friedreich ataxia (FRDA) is a progressive neurodegenerative disorder with limited treatments. Omaveloxolone, an Nrf2 activator, was approved based on a phase III trial showing modified Friedreich Ataxia Rating Scale (mFARS) improvement. Real-world and long-term data now require synthesis. To perform a single-arm meta-analysis pooling mean change in mFARS from baseline across all published omaveloxolone studies and interpret findings against natural history. Four studies (MOXIe Part 1, Part 2, open-label extension, Italian observational) comprising 248 patients were included. Data were harmonised using standard conversions. A random-effects model was used with subgroup and sensitivity analyses.The pooled mean ΔmFARS was −0.05 (95% CI −2.98 to 2.88; p=0.97). Subgroup analysis showed significant improvement in controlled trials (−2.19; 95% CI −3.12 to −1.27; p<0.00001) and consistent progression in real-world studies (+2.85; 95% CI 1.62 to 4.08; p<0.00001). Sensitivity analyses confirmed robustness. Controlled trials demonstrate significant short-term improvement with omaveloxolone, while real-world data show consistent progression at rates substantially slower than natural history. These complementary findings support a disease-modifying effect of omaveloxolone in FRDA.",
  "authors": [
    {
      "affiliations": [
        "Neurology Daepartment, Allied Hospital Faisalabad, Faisalabad, Punjab, Pakistan"
      ],
      "name": "Aasim Ali"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Waqar Ahmed Cheema"
    },
    {
      "affiliations": [
        "Neurology Daepartment, Allied Hospital Faisalabad, Faisalabad, Punjab, Pakistan"
      ],
      "name": "Fiza Nisar"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Ufaq Reyaz"
    },
    {
      "affiliations": [
        "Neurology Daepartment, Allied Hospital Faisalabad, Faisalabad, Punjab, Pakistan"
      ],
      "name": "Muhammad Saad"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Muhammad Talha"
    },
    {
      "affiliations": [
        "Neurology Daepartment, Allied Hospital Faisalabad, Faisalabad, Punjab, Pakistan"
      ],
      "name": "Sameen Sarfraz"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Ahmad Butt"
    },
    {
      "affiliations": [
        "Neurology Daepartment, Allied Hospital Faisalabad, Faisalabad, Punjab, Pakistan"
      ],
      "name": "Muhammad Shamoon"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Armaghana Abdullah"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Muhammad Asad Shabbir"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Muhammad Talha­"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Hammad Azam"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Muhammad Abdullah"
    },
    {
      "affiliations": [
        "Internal Medicine Department, Allied Hospital Faisalabad, Faisalabad, Pakistan"
      ],
      "name": "Aimen Anwaar"
    }
  ],
  "title": "Slowing the curve: a single-arm meta-analysis of mFARS outcomes following omaveloxolone treatment in Friedreich ataxia",
  "uid": "a4682470-f92a-5bca-b86e-daa1ed9e3276"
}