Quantitative nuclear magnetic resonance imaging detects subclinical changes over 1 year in skeletal muscle of GNE myopathy.
Laurent ServaisHarmen ReyngoudtJulien Le LouërAnthony BehinFerial ToumiMelanie VilleretEricky Caldas de Almeida AraujoPierre-Yves BaudinBenjamin MartyMelanie AnnoussamyJean-Yves HogrelPierre G CarlierLaurent ServaisPublished in: Journal of neurology (2019)
Even in a limited number of patients, qNMRI could detect a significant change over a 1-year period in GNE myopathy, which suggests that qNMRI could constitute a surrogate endpoint in this slowly progressive disease. Quantitative NMRI outcome measures can monitor intramuscular fat accumulation with high responsiveness. Longer follow-up should improve our understanding of GNE myopathy evolution and also lead to the identification of non-invasive outcome measures with the highest discriminant power for upcoming clinical trials.
Keyphrases
- magnetic resonance imaging
- skeletal muscle
- late onset
- clinical trial
- end stage renal disease
- ejection fraction
- newly diagnosed
- chronic kidney disease
- multiple sclerosis
- high resolution
- adipose tissue
- peritoneal dialysis
- prognostic factors
- computed tomography
- insulin resistance
- randomized controlled trial
- patient reported outcomes
- mass spectrometry
- type diabetes
- early onset
- magnetic resonance
- metabolic syndrome
- patient reported
- duchenne muscular dystrophy