A patient-centered qualitative evaluation of meaningful change on the NSAA and PUL in Duchenne Muscular Dystrophy
The perception of meaningful change in DMD as measured by the NSAA is influenced by ability levels and ambulatory function, with participants describing their need to maximize certain abilities, maintain function, and retain independence. For the PUL, participants underscored the importance of maintenance of their functional abilities, and highlighted key themes related to maintaining independence in ADLs, reaching, eating/drinking, and finger function for technology use across score categories.
Authors
- Teofil Ciobanu
- Krystian Przydzial
- Alexander P. Murphy (ORCID: https://orcid.org/0000-0003-4244-0541)
- Stefan Cano (ORCID: https://orcid.org/0000-0001-9390-273X)
- Jessica Mills
- Gerrit Vandenberg
- Arielle S. Gillman (ORCID: https://orcid.org/0000-0002-3511-4978)
- Johnson Alex (ORCID: https://orcid.org/0000-0002-8221-1383)
- Louise Barrett (ORCID: https://orcid.org/0000-0002-3299-4412)
- Phoebe Heinrich
- Bethany Ewens
- Evan Davies
- Anna Mayhew
Institutions
- Roche (Switzerland) (CH)
- Roche (United Kingdom) (GB)
- Duchenne UK (GB)
- Newcastle University (GB)
Publication Details
- Journal
- Frontiers in Neurology
- Published
- 2025-03-04
- DOI
- https://doi.org/10.3389/fneur.2025.1509174
- Citations
- 1
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81