Over £365,000 raised
so far !
The AADC Trust is launched 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 potentially incredible results and provided substantial relief from complex life-threatening symptoms for several AADCd children.
The AADC Research Trust is thrilled to report that Romain has received AAV2-hAADC Gene Replacement Therapy in Poland.
He is the fourth child to receive this treatment as a result of the #oneRAREstep Campaign, Romain's family say he is recovering well.
The AADC Research Trust wishes to thank all those who have supported the #oneRAREstep Campaign making it possible for Romain, Huzaifah, Marcus and Hallie to receive this incredible surgery.
There are still many more children suffering from this cruel disease so 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...
You can donate via our #oneRAREstep JustGiving main campaign page or, if there is a particular AADCd effected child that you would like to support, please select their individual personal appeals.