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Multimodal, Longitudinal Profiling of SCA1 Identifies Predictors of Disease Severity and Progression.

Teije H van ProoijeKirsten C J KapteijnsJack J A van AstenJoanna IntHoutMarcel M VerbeekTom W J ScheenenBart P C Van De Warrenburg
Published in: Annals of neurology (2024)
Our results guide the selection of the most sensitive measures of disease progression in SCA1 and have identified features associated with accelerated progression that could inform the design of clinical trials. ANN NEUROL 2024.
Keyphrases
  • clinical trial
  • genome wide
  • cross sectional
  • single cell
  • randomized controlled trial
  • dna methylation
  • phase ii
  • neural network
  • open label
  • phase iii