How families can participate in CDKL5 research
Hope through Action
Participation in CDKL5 research is critical for building a better future for our children and global CDKL5 community. There are several opportunities to get involved, from database registries and questionnaires to clinical trials and seeking care at a CDKL5 Center of Excellence.
We understand that living with CDKL5 disorder is overwhelming and often frightening, but we encourage you to find hope by engaging with the CDKL5 community. One of the most satisfying ways is by participating in research, no matter how small.
Everyone matters and can make a huge difference!
seeking your involvement!
CDKL5 Deficiency Disorder Database and Registry
The collection of data, clinical information and demographics of those affected by CDKL5 Disorder is vitally important to understanding the natural history of CDKL5 deficiency, and thereby helping to direct research and clinical efforts to find treatments and a cure.
We encourage every single family to consider participating in this effort. CDKL5 deficiency is a rare disorder, which means that every child's experience living with this condition needs to be recorded so that the community has a broad and full understanding of the spectrum of CDKL5 deficiency disorder.
We need your help!
Clinical Trial of Ganaxalone
by Marinus Pharmaceuticals
Marinus Pharmaceuticals has launched a pivotal trial of their drug Ganaxalone, a novel neurosteroid, for treatment of epilepsy in CDKL5 Deficiency Disorder (CDD). This global clinical trial is named the Marigold Study, and is now open for enrollment.