Every parent loves to watch their child fly like a butterfly and discover the world. Although five-year-old Ollie’s variation of CACNA1C means he is developmentally one year old, he plays... read more →
Shattering Rare Disease Registry Recruitment Targets with PIP-UK Real-world data from registries and other real-world evidence sources remain a fertile ground of opportunity for advocacy groups and biopharma companies to... read more →
A Collaborative Partnership to Develop the CDKL5 Registry CDKL5 Deficiency Disorder (CDD) is a rare developmental encephalopathy with epilepsy that is caused by mutations in the CDKL5 gene. CDD can... read more →
