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A longitudinal study of disease progression in facioscapulohumeral muscular dystrophy (FSHD).

Anika VarmaMichael S TodincaKaty EichingerSusanne HeiningerNuran DilekWilliam MartensRabi TawilJeffrey M StatlandJohn T KisselMichael P McDermottChad Heatwole
Published in: Muscle & nerve (2024)
The abilities and disease burden of adults with FSHD are largely static over a 12-month period with participants demonstrating a mild average reduction in some measures of strength. Selection of patients, outcome measures, and trial duration should be carefully considered during the design and implementation of future clinical studies involving FSHD patients.
Keyphrases
  • end stage renal disease
  • muscular dystrophy
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • peritoneal dialysis
  • prognostic factors
  • duchenne muscular dystrophy
  • patient reported
  • open label
  • current status