Sensitivity of Different Clinical Outcome Measures in Assessing Adults With Becker Muscular Dystrophy
RFFv was the only outcome measure sensitive to change at 3-year follow-up. Selecting on phenotype resulted in a high responsiveness for RFFv at 1-year follow-up and for 4SCv and NSAA at 2-year follow-up. NSAA could be performed in more participants compared with RFFv and therefore seems preferable in clinical trials for ambulant patients. Despite limitations in sample size, the results suggest that clinical trials in the ambulant population could be enriched by selecting on phenotype.
Authors
- Hermine A. van Duyvenvoorde (ORCID: https://orcid.org/0000-0003-0451-2065)
- E. Niks (ORCID: https://orcid.org/0000-0001-5892-5143)
- Jan J.G.M. Verschuuren (ORCID: https://orcid.org/0000-0002-4572-1501)
- Hermien E. Kan (ORCID: https://orcid.org/0000-0002-5772-7177)
- Melissa T. Hooijmans (ORCID: https://orcid.org/0000-0002-2233-1383)
- Iris Alleman
- Nienke M. van de Velde (ORCID: https://orcid.org/0000-0002-1541-130X)
- Jules J. van Benthem
- Zaïda Koeks (ORCID: https://orcid.org/0000-0003-4721-4965)
- Douwe E. Atsma (ORCID: https://orcid.org/0000-0002-9664-2985)
- Nina Ajmone Marsan (ORCID: https://orcid.org/0000-0001-7208-5769)
- Pietro Spitali (ORCID: https://orcid.org/0000-0003-2783-688X)
- E. Schrama (ORCID: https://orcid.org/0000-0003-3716-6584)
- Pieteke W. van Weperen
Institutions
- Utrecht University (NL)
- Leiden University Medical Center (NL)
- University Medical Center Utrecht (NL)
- Reinier de Graaf Hospital (NL)
- Duchenne Parent Project (NL)
- Amsterdam University Medical Centers (NL)
- Health & Safety in Sports (NL)
Publication Details
- Journal
- Neurology
- Published
- 2025-09-05
- DOI
- https://doi.org/10.1212/wnl.0000000000214071
- Citations
- 1
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81