Evaluation of a serum protein signature as monitoring biomarker for Duchenne Muscular Dystrophy in a long-term clinical trial with corticosteroids
These findings support the utility of serum protein signatures as objective, quantitative tools for monitoring disease progression and treatment response in DMD during clinical visits and clinical trials.
Authors
- Y. Van Der Burgt
- Michela Guglieri (ORCID: https://orcid.org/0000-0002-8455-0637)
- Rebecca Tobin (ORCID: https://orcid.org/0000-0001-7918-3193)
- Sharon de Vries
- Albert Jiménez-Requena
- Utkarsh J. Dang (ORCID: https://orcid.org/0000-0003-4120-2015)
- C. Degan (ORCID: https://orcid.org/0009-0001-9993-9346)
- Pietro Spitali (ORCID: https://orcid.org/0000-0003-2783-688X)
- Amela Peco
- Jordi Diaz-Manera
- Roula Tsonaka
- FOR-DMD investigators of the Muscle Study Group
- Yetrib Hathout
- Cristina Al-Khalili Szigyarto
- Bart J. M. Vlijmen
Institutions
- Binghamton University (US)
- Leiden University Medical Center (NL)
- Hospital de Sant Pau (ES)
- Centro de Investigación Biomédica en Red (ES)
- Muscular Dystrophy UK (GB)
- Carleton University (CA)
- KTH Royal Institute of Technology (SE)
Publication Details
- Journal
- medRxiv
- Published
- 2026-01-02
- DOI
- https://doi.org/10.64898/2025.12.18.25342544
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Parent Project Muscular Dystrophy
- Leids Universitair Medisch Centrum
- National Institutes of Health