The Facioscapulohumeral Muscular Dystrophy Rasch‐Built Overall Disability Scale (<scp>FSHD</scp>‐<scp>RODS</scp>): Longitudinal Assessment of a Disease‐Specific Patient Reported Outcome
FSHD progression is generally slow, with FSHD-RODS only detecting meaningful decline in moderately to severely affected patients. The ability to compensate for muscle weakness may limit the sensitivity of patient-reported outcomes like FSHD-RODS in clinical trials. These findings highlight the need for alternative measures to capture disease progression and treatment effects in FSHD research.
Authors
- Sjan Teeselink (ORCID: https://orcid.org/0000-0003-1023-2182)
- Nicol Cornelia Voermans (ORCID: https://orcid.org/0000-0002-5837-7295)
- Sanne C. C. Vincenten (ORCID: https://orcid.org/0000-0002-0412-0670)
- Karlien Mul (ORCID: https://orcid.org/0000-0001-8487-9478)
- Baziel G.M. van Engelen (ORCID: https://orcid.org/0000-0001-9867-9047)
- Nens van Alfen (ORCID: https://orcid.org/0000-0001-7839-8125)
Institutions
- Radboud University Nijmegen (NL)
- Radboud University Medical Center (NL)
Publication Details
- Journal
- European Journal of Neurology
- Published
- 2025-08-01
- DOI
- https://doi.org/10.1111/ene.70238
- Citations
- 2
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.61
Funders
- Prinses Beatrix Spierfonds