#oneRAREstep

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:

Breaking News...

The AADC Research Trust is thrilled to report that, as a result of the #oneRAREstep Campaign, our first child suffering from AADCd has received AAV2-hAADC Gene Replacement Therapy in Poland. 

Hallie's family say she is recovering well.

 

The AADC Research Trust wishes to thank all those who have supported the #oneRAREstep Campaign making it possible for 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...

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How to get involved...

JustGiving...

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 Hallie becomes the first AADCd child, participating in our #oneRAREstep Campaign, to receive AAV2-hAADC Gene Replacement Therapy in Poland. So far this year Hallie, Haia, Rian, Aimar, Kacper, Ania, Irai and Ted have all received this potentially lifesaving treatment. This now makes a total of 8 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:

Hallie
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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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

Gene Therapy Trials: USA

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