Our Impact
We offer families immediate relief, provide essential education, and bring hope when there is nowhere else to turn. From crucial wound care supplies to access to an on-call nurse, we are a lifeline to thousands of families nationwide.
Some families are facing medical supply bills of $80,000 per month. We do everything possible to alleviate this burden and spend more than $1.5 million dollars on direct-to-patient programs and services every year.
Because this is the worst disease you’ve never heard of, we partner closely with academic researchers, elected officials on Capitol Hill, the U.S. FDA, and the drug development industry to drive meaningful change for the patient community.