Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy.
Hiroyuki AwanoYoshinori NambuChieko ItohAkihiro KidaTetsushi YamamotoTomoko LeeYasuhiro TakeshimaKandai NozuMasafumi MatsuoPublished in: Muscle & nerve (2024)
We found that creatine kinase levels and motor, respiratory, and cardiac functions each exhibited various changes over time. These findings provide useful information about the longitudinal data of several outcome measures for patients with DMD not receiving corticosteroids. These data may serve as historical controls in comparing the natural history of DMD patients not on regular steroid use in appropriate clinical trials.
Keyphrases
- duchenne muscular dystrophy
- electronic health record
- clinical trial
- end stage renal disease
- big data
- left ventricular
- muscular dystrophy
- chronic kidney disease
- ejection fraction
- newly diagnosed
- tyrosine kinase
- protein kinase
- heart failure
- healthcare
- randomized controlled trial
- peritoneal dialysis
- artificial intelligence
- open label
- phase ii
- deep learning
- placebo controlled