{
  "abstract": "Objectives To investigate the feasibility of remote assessments of patient-reported outcomes (PRO) and utility of digital biomarkers in patients with spinocerebellar ataxia (SCA) and primary mitochondrial disease (PMD).Method The study is currently recruiting adult patients with confirmed genetic diagnoses of PMD (n=20) and SCA (n=40). The following assessments are completed at the baseline visit, followed by two monthly remote digital assessments for up to a year.• Clinician-reported outcome measures (CRO): 9-hole Peg Test, SARA (Scale for Assessment and Rating of Ataxia, Inventory of Non-Ataxia Signs, Mini-Best (Balance Evaluation Systems Test), and Newcastle Mitochondrial Disease Adult Scale• Patient reported outcome measures (PRO): Activities-specific Balance Scale, Hospital Anxiety and Depression Scale• Digital Assessments: Patient-reported outcome measure of Ataxia (PROM-Ataxia), Activities of Daily Living, Components of the Quality of Life in Neurological Disorders, Cognitive-Motor Tasks and Speech Assessment tasks; the digital platforms are supported by the Neurosciences team at Monash University, Australia (led by Prof I Harding).Results Preliminary findings show moderate to strong correlations between SARA and remote assessment tasks. This project offers a research opportunity to patients with rarer genetic aetiologies who have more advanced symptoms and explore the practicality of remote assessments in overcoming access barriers to research.Teddymoe29@gmail.com",
  "authors": [
    {
      "affiliations": [
        "NIHR Newcastle Biomedical Research Centre, Newcastle University",
        "Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute, Newcastle",
        "NHS Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle,UK",
        "Directorate of Neurosciences, Royal Victoria Infirnary, Newcastle,UK"
      ],
      "name": "Moe Aye M"
    },
    {
      "affiliations": [
        "School of Psychological Sciences, Monash University, Melbourne, Australia"
      ],
      "name": "Selvadurai Louisa P"
    },
    {
      "affiliations": [
        "NIHR Newcastle Biomedical Research Centre, Newcastle University",
        "Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute, Newcastle",
        "NHS Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle,UK",
        "Directorate of Neurosciences, Royal Victoria Infirnary, Newcastle,UK"
      ],
      "name": "Newman Jane"
    },
    {
      "affiliations": [
        "NIHR Newcastle Biomedical Research Centre, Newcastle University",
        "Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute, Newcastle",
        "NHS Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle,UK",
        "Directorate of Neurosciences, Royal Victoria Infirnary, Newcastle,UK"
      ],
      "name": "Barrow Isabel"
    },
    {
      "affiliations": [
        "Cerebellum and Neurodegeneration Research Group, QIMR Berghofer Medical Research Institute"
      ],
      "name": "Harding Ian"
    },
    {
      "affiliations": [
        "NIHR Newcastle Biomedical Research Centre, Newcastle University",
        "Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute, Newcastle",
        "NHS Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle,UK",
        "Directorate of Neurosciences, Royal Victoria Infirnary, Newcastle,UK"
      ],
      "name": "Gorman Grainne S"
    },
    {
      "affiliations": [
        "NIHR Newcastle Biomedical Research Centre, Newcastle University",
        "Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute, Newcastle",
        "NHS Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle,UK",
        "Directorate of Neurosciences, Royal Victoria Infirnary, Newcastle,UK"
      ],
      "name": "Ng Yi S"
    }
  ],
  "title": "98 R-PROMS: a feasibility study of remote patient reported outcome measures in patients with ataxia",
  "uid": "5360d6c4-09b1-5cef-9f92-db8348751909"
}