Publication: Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: A qualitative study and the development of a patient-centered conceptual model

This research article, published in the Journal of Patient Reported Outcomes, describes the development of a conceptual model of the symptoms and impacts of nonsense mutation Duchenne muscular dystrophy (nmDMD), based on qualitative interviews with caregivers

  • Review the process used to undertake a qualitative study involving interviews with 10 caregivers of patients with nmDMD in the UK
  • Discover the symptoms and impact of nmDMD before and after treatment with a mutation-specific therapy, as reported by caregivers
  • Learn about a conceptual model, illustrating the relationships between symptoms and impacts of nmDMD before treatment

Williams K, Davidson I, Rance M, et al. J Pat-Rep Outcomes. 2021;5:75

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