If you or a loved one are affected by PCDH19 Epilepsy you can join our Facebook page for updates on ongoing PCDH19 research. This group is an international information group co-run by The Cute Syndrome and our partners in Spain and France. Click on the link above and request to join the group. We will respond to confirm (check your "Other" for our message. Otherwise, email me directly to be admitted.
Marinus Pharmaceuticals announces a plan for initiating an open-label Phase 2 proof-of-concept clinical trial under an expanded access protocol under our epilepsy IND in up to 10 patients with PCDH19 female pediatric epilepsy. Visit Marinus Pharmaceuticals for more information.
RARE Toolkits provide individuals with usable information on a variety of topics related to living with and/or advocating for rare disease patients. RARE Toolkits are being created in collaboration with key rare disease stakeholders that have developed a vast array of subject matter expertise and believe in the importance of sharing these best practices. RARE Toolkits are interactive and will continue to evolve as advocates share additional best practices around specific topics. These important resources are being made available online, as a print version that can be downloadable in a PDF format, and in print.