#oneRAREstep Campaign


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...


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.  

Social Media...

For more information and updates on the Campaign please follow us on our Social Media platforms...

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quick links

stay in touch

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find us

e: enquiries@aadcresearch.org

Tel: 01883 674467


The AADC Research Trust

Soper Hall, 2 Harestone Valley Road,

Caterham, Surrey, CR3 6HY

Registered Charity UK & Wales No: 1114367

© AADC Research Trust