Finding suitable clinical endpoints for a potential treatment of a rare genetic disease: the case of arid1b

HIGHLIGHTS

  • who: Matthijs D. Kruizinga from the Centre for Human Drug Research CL Leiden, the Netherlands Department of Clinical Genetics, Leiden University Medical Centre have published the paper: Finding Suitable Clinical Endpoints for a Potential Treatment of a Rare Genetic Disease: the Case of ARID1B, in the Journal: (JOURNAL)
  • what: The aim of this study is to explore a novel approach towards developing new endpoints for neurodevelopmental disorders in this case for ARID1B-related ID.
  • future: While the included tests have at least a theoretical relationship between disease severity and test outcome as outlined . . .

     

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