Advancing Outcome Measures for SCN8A and Other Severe DEEs

Through our DEE-P Connections partnership, we are pleased to be supporting an important collaborative effort to advance improved biomarkers and outcome measures for those severely affected by SCN8A and other DEEs and neurodevelopmental disorders.

One of the many distinct challenges of severely affected DEEs is the absence of validated biomarkers and outcome measures (BiOMes). Measurement of skills and functions is essential for:

  • Evidence-based treatment and CARE
  • Capturing small but valued improvements during CLINICAL TRIALS
  • RESEARCH – to increase the depth and breadth of understanding of conditions and changes over time of vital life features of severe DEEs.

DEE-P Connections developed a partnership with a consortium of rare developmental disorders focused on developing biomarkers and outcome measures. Our effort brings together a highly skilled research team, 10 committed patient group leaders, and diverse experts working on improved outcome measures for those severely affected by DEEs and other neurological disorders.