Hear from the experts: Importance of Quality of Life in Patients with DMD

Duchenne muscular dystrophy (DMD) places a substantial burden on the everyday lives of patients and families.1–3 While preserving mobility and independence is a priority, is there enough focus on whether the existing quality of life (QoL) metrics are specific enough for DMD? Should we look at QoL differently? Or at least tailor our approach to this unique patient population?

In this webinar, gain insights and knowledge about the changing landscape of QoL in DMD from a panel of experts.

Watch now to find out more about:

  • The importance of health-related quality of life (HRQoL) in DMD
  • The impact of DMD on HRQoL at different stages of ambulation
  • How HRQoL is currently measured in DMD patients
  • Best practice to support DMD patients and protect their QoL
  • Insights from real-life patient cases 

Dr Sarah Acaster (Acaster Lloyd Consulting Ltd, London, UK) shares her insights on HRQoL in DMD based on her extensive knowledge of qualitative research in the area, and reports on the findings of a recently published study that help to define what it actually means to be an individual with nonsense mutation DMD (nmDMD) or the caregiver of an individual with nmDMD.

Dr Erik Landfeldt (IQVIA, Stockholm, Sweden) explores the variety of tools and scales available to measure QoL, giving a balanced and in-depth view of their suitability for use in children with DMD.  

Dr Edicson Ruiz (Professor of Child Rehabilitation, Universidad Nacional de Colombia, Bogotà, Columbia) presents a detailed case study of a young patient with DMD, looking at the challenges they have to overcome and the impact these challenges have on perceived QoL.

The webinar was organised, funded and hosted by PTC Therapeutics.

1. Landfeldt E, et al. J Neurol. 2016;263:906–915.
2. Landfeldt E, et al. Acta Paediatrica. 2019;108:224–230.
3. Ryder S, et al. Orph J Rare Dis. 2017;12:79.

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