Gene Therapy Treatment Facts...
This pioneering surgery delivers an AAV2-hAADC virus vector in a specially designed cannula to the Substantia Nigra Pars Compacta and Ventral Tegmental area of the brain. During the process real time MRI imaging of the patients brain is used to map the target region, plan cannula insertion and positioning, and calculate how much of the vector to infuse. It takes approximately 8 hours to complete.
Pre and post operative Care:
Pre and post operative tests include PET scans, lumbar punctures, DNA sequencing tests, blood tests, enzyme assays and MRIs.
The surgery is performed by a team of four neurosurgeons lead by Professor Krystof Bankiewicz and Professor Miroslaw Zabek.
The Interventional Neurotherapy Center (INC), Brodno Hospital, Warsaw, Poland.
The AADC Trust's Role
Managing Patient Expectations:
The AADC Trust is a vital partner for families accessing this treatment, bridging the gap between patients and professionals, and guiding them throughout this journey.
Funding will be sourced through various methods - philanthropy, public funding and health authorities. Once funding has been secured the Trust will assist families with the logistical process and provide ongoing support throughout their child's Gene Therapy journey. We can provide information with suggestions for hospital and nearby accommodation, local travel, places of interest and translation assistance (if required). Please contact at
Managing Professional Expectations:
The AADC Trust collaborates with worldwide medical and scientific AADCd experts who can assist professionals, unfamiliar with this ultra-RARE brain disease, with the care of their patients. We will ensure professionals have clear Gene Therapy 'Perioperative' guidelines and will be teamed with an AADC expert affiliated to the treatment if necessary.
AADCd children have been treated with AAV2-hAAD in
have been treated with AVV2-hAADC
will be treated in Poland over the next 18 months on a 'not-for-profit basis.
more children will be treated in the USA over the next 18 months on the 'Extended Single-Stage Trial'
mutations have been discovered on the DDC gene
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.
Disclaimer: The AADC Trust reserves the right that in the event it successfully raises the required funds or acquires government funds from the country where a child resides to cover the treatment, it will use any surplus monies raised, from their personal page, to assist with their off treatment costs, such as travel and accommodation. Any remaining funds will be used by the Trust for the purpose of this Campaign, including distributing them to other children registered for the treatment.