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Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints.

Alexander Peter MurphyJasper MorrowJulia Rebecka DahlqvistTanya StojkovicTracey A WillisChristopher D J SinclairStephen WastlingTarek YousryMichael G HannaMeredith K JamesAnna G MayhewMichelle EagleLaurence E LeeJean-Yves HogrelPierre G CarlierJohn S ThorntonJohn VissingKieren G HollingsworthVolker Straub
Published in: Annals of clinical and translational neurology (2019)
These results further support fat fraction measurements as a primary outcome measure alongside functional assessments. The most appropriate individual muscles are the vastus lateralis, gracilis, sartorius, and gastrocnemii. Using composite groups of lower leg muscles, thigh muscles, or triceps surae, yielded high standardized response means (SRMs). Over 6 years, quantitative fat fraction assessment demonstrated higher SRM values than seen in functional tests suggesting greater responsiveness to disease progression.
Keyphrases
  • muscular dystrophy
  • adipose tissue
  • duchenne muscular dystrophy
  • clinical trial
  • fatty acid
  • study protocol
  • high resolution
  • phase iii
  • phase ii
  • open label
  • mass spectrometry