A 5‐year natural history cohort of patients with facioscapulohumeral muscular dystrophy determining disease progression and feasibility of clinical outcome assessments for clinical trials
The captured FSHD disease progression rate in 5 years was generally minimal. The COAs in this study are not feasible for clinical trials with a duration of 2 years. Extended trial durations or novel outcome assessments might be necessary to improve trial feasibility in FSHD.
Authors
- Joost Kools (ORCID: https://orcid.org/0000-0002-6838-9345)
- 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)
- Nicoline Voet (ORCID: https://orcid.org/0000-0001-7381-2817)
- Corinne G.C. Horlings (ORCID: https://orcid.org/0000-0002-2440-9180)
- Baziel G.M. van Engelen (ORCID: https://orcid.org/0000-0001-9867-9047)
- Ingemar S. J. Merkies (ORCID: https://orcid.org/0000-0002-8516-2361)
Institutions
- Innsbruck Medical University (AT)
- Radboud University Nijmegen (NL)
- Universität Innsbruck (AT)
- Maastricht University Medical Centre (NL)
- European Association of Urology (NL)
- Radboud University Medical Center (NL)
- Maastricht University (NL)
- University of Curaçao (CW)
Publication Details
- Journal
- Muscle & Nerve
- Published
- 2024-11-07
- DOI
- https://doi.org/10.1002/mus.28293
- Citations
- 6
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.40
Funders
- Prinses Beatrix Spierfonds