The AADC Trust is launching the #oneRAREstep Campaign to raise funds for our AADCd children to access potentially life saving and disease transforming, pioneering brain delivered AAV2-hAADC Gene Therapy Treatment.
We are walking our way to a CURE with a ground breaking treatment available to our
children; AAV2-hAADC Gene Replacement Therapy. The surgery that is performed in Poland is based on GT approach developed under NIH grant, approved by the FDA, 2 US IRB's and constitute experimental treatment to save lives of AADC deficient children. It is setting a benchmark for future treatments and has the potential to help many other children suffering from
diseases with single gene defects.
AAV2-hAADC Gene Replacement Therapy has already shown incredible results and provided substantial relief from complex life-threatening symptoms for several AADCd children.
Individual #oneRAREstep JustGiving Fundraising pages
Please click on the images below to read their stories and donate to help them raise funds for this potentially life saving and transformative treatment...
How to get involved...