In a crucial advancement towards tackling CADASIL, a rare genetic disease, the CADANHIS project has been launched with significant financial support from the European Joint Programme on Rare Diseases (EJP RD).
CADASIL, characterized by recurrent strokes, progressive cognitive decline, and adult-onset disability, is linked to stereotyped mutations of the NOTCH3 gene. Unfortunately, there is currently no specific therapy for this severe condition.
The CADANHIS initiative marks a significant collaborative effort, bringing together patients, families, and expertise from five European countries. The project’s diverse goals include improving understanding, developing patient-reported outcomes, identifying relevant clinical results, and discovering biomarkers associated with CADASIL.
Employing cutting-edge methodologies, CADANHIS will examine years of clinical and imaging data, enriched by insights from patients and their families. The project expects to generate a significant collection of findings, data, and resources crucial in the pursuit of developing targeted treatments for CADASIL.
The primary objective is to deliver the right treatment to the right patient at the right time. This innovative initiative holds the potential to make significant progress in addressing the challenges presented by CADASIL, thereby improving the lives of those affected by this rare genetic disease.