RESEARCHERSInformation & opportunities to advance research
Our work includes:
- Raising awareness among physicians, scientists, and the general public;
- Supporting relevant research through an international grant research program;
- Providing a caring and educational community for affected families; and
- Hosting a unique information resource.
Working together we are making a difference in the lives of children and their families. One day there will be a cure; we hope you will help us make that day come sooner.