Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Congress activities | Academy of Managed Care Pharmacy (AMCP) | AMCP 2021 poster: Symptoms and impacts of nonsense mutation Duchenne muscular dystrophy (nmDMD): A qualitative study and the development of a patient-centered conceptual model

AMCP 2021 poster: Symptoms and impacts of nonsense mutation Duchenne muscular dystrophy (nmDMD): A qualitative study and the development of a patient-centered conceptual model

This poster, shown at the 2021 virtual AMCP Annual Meeting, presents results of a qualitative interview study investigating the impact on health-related quality of life (HRQoL) of nmDMD, and patients’ and caregivers’ experience of treatment

  • Review the demographics of 10 caregivers who participated in the interviews, and characteristics of the ambulatory patients with nmDMD
  • Explore the symptoms and the impact on HRQoL of nmDMD before and after treatment with a mutation-specific DMD therapy, as reported by caregivers
  • Understand the experience of patients and caregivers with the mutation-specific DMD therapy
  • Learn about a conceptual model of nmDMD, including moderating factors and management, developed from the responses

▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC at pharmacovigilance@ptcbio.com

Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.

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MED-ALL-DMD-2200031 | March 2022
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