CANDID Study (hosted by the LouLou Foundation in coordination with seven biopharmaceutical industry partners)

The CANDID (Clinical Assessment of NeuroDevelopmental measures  In CDD) is a three year observational study which investigates the baseline and longitudinal performance of known and validated instruments and scales, measuring cognitive, motor, and developmental domains in up to 100 CDD patients. CANDID will provide researchers with information on appropriate tests evaluating disease-modifying therapeutics for CDD, such as gene therapies, enzyme replacement, genome editing, or small molecules. CANDID study results will eventually be shared with the entire community to aid CDD clinical trial design and inform therapeutic development for related neurodevelopmental disorders.

This study is now underway in Canada and at sites around the world. CDKL5 Canada is proud to be supporting this effort.

Ultragenyx  UXO55 – Investigational Gene Therapy Program for CDD

We are pleased to share with you the UX055 Patient Advocacy Update from Ultragenyx. Please click here to view the full letter from Kristin Voorhees, Director of Patient Advocacy. Their team is working hard on our children’s behalf and we are so happy to share their progress with you!

Be the first to find out about more opportunities like this in Canada, by joining our Canadian registry, Link Canada.