Old Globe

International Patient Organisation for the ultra RARE disease; Aromatic Amino Acid

Decarboxylase deficiency(AADCd)

Celebrating 15 YEARS 

Latest Gene Therapy News ...

This year has brought many challenges with the emergence of COVID-19. Many aspects of our lives have been put on hold.


Essential surgeries, including AAV2-hAADC Gene Therapy Treatment, had to be postponed due to the extremely difficult circumstances posed by the threat of COVID.


However, Professor Bankiewicz and his incredible team have gone to extraordinary lengths to meet that challenge...


The AADC Trust is thrilled to share the wonderful news that three more AADCd children have received Gene Therapy. Two received treatment in Poland and one received treatment in the USA ...

Meet 11 year old Julia from Norway. Julia is the 21st AADCd child to receive

AAV2-hAADC Gene Replacement Therapy.

Meet 10 year old Paul from Germany. Paul is the 22nd AADCd child to receive

AAV2-hAADC Gene Replacement Therapy.

Meet 6 year old Jamell from the USA.

Jamell is the 23rdd AADCd child to receive

AAV2-hAADC Gene Replacement Therapy.

There are still many more children suffering from this cruel disease, waiting to access this potentially life saving and disease transforming surgery. Please read below to see how you can help... 


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 potentially incredible results and provided substantial relief from complex life-threatening symptoms for several AADCd children.

Breaking News...

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.

Individual #oneRAREstep JustGiving Fundraising pages:

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.  

Social Media...

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

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About AADC deficiency...

Aromatic Amino Acid Decarboxylase deficiency (AADCd) is an Ultra-RARE autosomal recessive neurometabolic ‘Parkinsonism’ disorder causing an enzyme deficiency primarily affecting the conversion of L-dopa to Dopamine and 5HTP to Serotonin.  The deficiency of these essential neurotransmitters has catastrophic consequences resulting in life-limiting, life-threatening and multiple disabilities.  The disease currently has a world incidence of 1:55,000,000.  Approximately 150-200 known sufferers are said to span more than 30 countries.  Every year children and young adults lose their lives to this brain disease. The AADC Research Trust, is the only International dedicated patient group committed to improving treatments and discovering a cure.

Exciting Medical Breakthrough for the Treatment of AADC deficiency...

Follow our Children's AAV2-hAADC Gene Therapy Journey

LANDMARK moment as Romain, aged 16, from the France, has now received AAV2-hAADC Gene Therapy Treatment in Poland. He is the 3rd young ADULT to receive the pioneering brain surgery.

Teenagers and young adult AADCd sufferers often develop bone deformities, such as scoliosis and contractures, as a consequence of many years of suffering repeated oculogyric crises (OGC’s). Assuming our children survive into adulthood, at some stage they will likely need corrective surgery, offering only short term relief, as their OGC’s will continue and so the cycle will begin again. However, Gene Therapy, is proving to lead to a cessation of OGC’s, which is hugely SIGNIFICANT to future corrective surgeries that these young adults may need. Gene Therapy not only makes them strong enough to withstand such surgery, it also means that it will be a long-term sustainable solution, as the OGC’s are no longer a cause for concern. 

So far this year Paul, Julia, Romain, Yusuf, Huzaifah, Marcus, Swathi, Hallie, Haia, Rian, Aimar, Kacper, Ania, Irai and Ted have all received this potentially lifesaving treatment. This now makes a total of 15 AADCd sufferers receiving this pioneering brain surgery in EUROPE courtesy of Professor Krystof Bankiewicz and his incredible medical team, with more children scheduled in the coming months...

Click on the images to discover more about this pioneering and transformative treatment:

Latest AADCd News...
Keeping up to date with the latest developments

Essential Disease Links...

Gene Therapy Trials: USA
Gene Therapy Treatments: Poland
Columbus Children's Founsation
Gene Therapy Statistics
Gene Therapy Trials: Japan/Taiwan
Genetic Variations in AADCd: Italy
AADCd Stem Cells: UK
AADCd Awareness Week 2019
Treatment Guidelines: English
AADCd World Conference UK 2018
Treatment Guidelines: Chinese
AADCd China Conference 2019
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Explore our links for essential information on AADCd...

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The iNTD Database and Patient Registry
The iNTD Database is an anonymous database which created the AADCd Consensus Guideline for the diagnosis and management of the disease. If you have a child with AADCd please register them to the iNTD Database....
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Consensus Guidelines Publication - iNTD
Click on the images if you would like more information on the iNTD Registry
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Follow our amazing AADCd Warriors on Social Media...

Click on the images to go to our "Follow our children on Social Media" page...

Supporting AADCd Families...

Click on the links for useful information for recently diagnosed AADCd families...

Siblings AADCd Story Book
Get Involved
Facebook Family Support Group
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To contact us please email via: enquiries@aadcresearch.org and we will endeavour to reply to you as soon as possible. We apologise in advance for any delays in our response, during this time. Our staff are on furlough and working from home. Please bear with us. Thank you for your patience.


The AADC Research Trust

Soper Hall, 2 Harestone Valley Road,

Caterham, Surrey, CR3 6HY

Registered Charity

UK & Wales No: 1114367

© AADC Research Trust

Facebook Family Support Group

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