Right now, there is no cure—but there is hope.

Science is moving quickly and treatments have been developed that can slow down the progression of the disease, and we’re making sure that progress reaches families faster.

We fund promising science, build clinical trial capacity, and empower families with knowledge and support.

The money that we invest in research has been raised by a community of fundraisers and donors. By working with researchers, clinicians, and industry partners, we make sure this money is strategically targeted at bringing us closer to new treatments.