Advancing Outcome Measures for SCN8A and Other Severe DEEs
Through our DEE-P Connections project, we are pleased to be leading an important collaborative effort, The Inchstone Project, 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 disorders like SCN8A, with a large population who are severely affected by the disease, is the absence of validated outcome measures to measure abilities, skills and functions which 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.disorder
DEE-P Connections developed a partnership with a consortium of rare developmental disorders focused on developing outcome measures for those most profoundly impacted by disorders such as SCN8A. Our effort brings together a highly skilled research team, committed patient advocacy groups, pharmaceutical partners aand diverse experts working on improved outcome measures for those severely affected by DEEs and other neurological disorders.
