Lessons for future clinical trials in adults with Becker muscular dystrophy: Disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures.
Bram De WelLouise IterbekeLotte HuysmansRonald PeetersVeerle GoosensNicolas DubuissonPeter Y K Van den BerghVinciane Van ParijsGauthier RemicheLiesbeth De WaeleFrederik MaesPatrick DupontKristl G ClaeysPublished in: European journal of neurology (2024)
It is concluded that proton density fat fraction imaging of entire thigh muscles is a sensitive outcome measure to track progressive muscle fat replacement in patients with BMD, already after 9 months of follow-up. Finally, significant changes are reported in a wide range of clinical and patient-reported outcome measures, of which the MFM-32 appeared to be the most sensitive to change in adults with BMD.
Keyphrases
- patient reported
- muscular dystrophy
- magnetic resonance imaging
- clinical trial
- adipose tissue
- skeletal muscle
- multiple sclerosis
- high resolution
- duchenne muscular dystrophy
- computed tomography
- fatty acid
- randomized controlled trial
- current status
- magnetic resonance
- open label
- mass spectrometry
- phase ii
- study protocol
- diffusion weighted imaging