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International Patient Organisation for the ultra RARE disease; Aromatic Amino Acid
Decarboxylase deficiency (AADCd)
A monumental day for the AADCd community as NICE approves the gene therapy drug Upstaza for use in the UK. This remarkable milestone brings hope to many children living with rare diseases ...
The AADC Research Trust is an International Patient Advocacy Organisation, representing children suffering from the ultra rare brain disorder Aromatic Amino Acid Decarboxylase deficiency; AADCd. Read more about us & the critical work we do ...
Discover the incredible work we are doing to drive critical disease research, advance treatment strategies, improve the diagnostic odyssey with simplified testing & much more ...
Browse this section to view the many International AADCd Medical & Scientific Conferences held by The Trust. Watch our much anticipated AADCd Family Workshop videos with transcripts in several languages ...
We are at the forefront of pioneering Gene Therapy treatments. Discover more about these incredible treatments, our involvement in driving them forward & follow the journeys of our AADCd children who have already had AAV2-AADC Gene Therapy ...
Everything you need to know as family of a child suffering from AADCd. Find out how to recognise signs & symptoms, what to do next, what support is available and much more ...
There are many ways you can get involved & support us as we continue to raise global awareness of this devastating disease. Find out how you can help us change the lives of our amazing AADCd warriors ...
For all enquiries please email us at enquiries@aadcresearch.org