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A Cross-Sectional Study of Nemaline Myopathy.

Kimberly AmburgeyMeryl AckerSamia SaeedReshma AminAlan H BeggsCarsten G BonnemannMichael BrudnoAndrei ConstantinescuJahannaz DastgirMamadou DialloCasie A GenettiMichael GlueckStacy HewsonCourtney HumMinal S JainMichael W LawlorOscar H MeyerLeslie L NelsonNicole SultanumFaiza SyedTuyen TranChing H WangJames J Dowling
Published in: Neurology (2021)
We present a comprehensive cross-sectional study of NM. Our data identify significant disabilities and support a relatively stable disease course. We identify a need for further diagnostic investigation for the genetically unresolved group. MFM, PFTs, and the slurp test were identified as promising outcome measures for future clinical trials.
Keyphrases
  • clinical trial
  • electronic health record
  • late onset
  • physical activity
  • photodynamic therapy
  • current status
  • big data
  • phase ii
  • randomized controlled trial
  • early onset
  • phase iii
  • deep learning
  • double blind
  • data analysis