Our Research Mission
Cure CLCN4 was founded with the goal of advancing the much-needed scientific research on CLCN4-related condition. It is built on the foundation of hope and commitment, that one day there would be a cure for Daphne and children like her.
We aim to do this by…
- Providing the scientific and clinical community with the necessary research tools to advance our understanding of CLCN4-related condition.
- Identifying and funding research projects with the potential to contribute to current knowledge of the biology and pathomechanisms involved in CLCN4-related condition.
- Helping establish an international network of researchers and clinicians involved in research on CLCN4-related condition and/or the management of individuals with this condition.
- Supporting the identification of patients with CLCN4-related condition by working with genetic laboratories, both in the UK and abroad, to add CLCN4 to genetic testing panels and providing families with available resources.
- Support efforts to establish and maintain an official CLCN4 patient registry, with the ultimate goal of building a natural history data set.
- Raise awareness of CLCN4-related condition among the scientific and medical community and promote research engagement with this condition.
“Ultimately, our objective is to bring together patients, researchers, clinicians, & biomedical industry in a shared mission to improve our understanding of CLCN4-related condition, with the aim of developing breakthrough therapies that improve the quality of life of those living with this condition”