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249th ENMC International Workshop: The role of brain dystrophin in muscular dystrophy: Implications for clinical care and translational research, Hoofddorp, The Netherlands, November 29th–December 1st 2019

Published:August 14, 2020DOI:https://doi.org/10.1016/j.nmd.2020.08.357

      Highlights

      • Overview of the multiple dystrophin isoforms expressed in the brain.
      • Large body of preclinical data and possibilities for postnatal dystrophin restoration.
      • Overview of neurocognitive and behavioral heterogeneity in DMD and BMD.
      • Neuromuscular specialists to also focus on early diagnosis and intervention for brain comorbidities.
      • Need to develop standard operation procedures for neurocognition and behavior assessment.

      Keywords

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