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4th AADCd World  Conference - 2018

The AADC Research Trust would like to thank everyone who attended our 4th World Conference this year at the stunning De Vere Wotton in Dorking. Your incredible contributions were truly appreciated. It was an extremely informative conference, with lots of high level science discussed, including great therapeutic interventions coming forward such as Gene Therapy becoming available to the masses. Our AADC families were given the rare opportunity to connect with professionals and leading experts from around the world. We are looking forward to seeing how these important discussions will advance our ultimate aim of finding a cure.


A Welcoming Introduction to our 4th International AADCd Conference

An Overall Picture of the Structure-Function Relationship of AADC Variants Causing AADC Deficiency
CSF Findings in a Carrier of AADC Deficiency/
Urinary Sulphatoxymelatonin
Probiotics in DDC Mouse
Update on MRI-guided midbrain Gene Therapy Trial in Children with AADCd

Non-Invasive PET Visualisation of AADCd Manifestations. Therapy Planning. Outcomes. Monitoring and Theranostics/Artificial Intelligence for Underserved and Rare Paediatric Conditions

Marcel Verbeek ISAO Nijmegen 0216 HIGH R
Next Generation Metabolic Screening for Elucidation of AADC deficiency and Related Neurometabolic Disorders

Dysonia Scale in Paediatric Neurotransmitter Disorders: A Pilot Study

Conference Chair & Biochemical Introduction to AADCd
Professor Marcello Coba
Modelling Human Disease with iPSC Derived Neurons
Successful Pregnancy in a Patient with L-Amino Acid Decarboxylase Deficiency: Therapeutic Management & Clinical Outcomes
Natural History Study AADCd: A Retrospective Study
Gene Therapy Case Studies in Japan
Next Generation Metabolic Screening for Elucidation of AADC deficiency and Related Neurometabolic Disorders
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Measuring the Burden of Illness in AADCd
A Clinical Introduction to AADCd 
Generation of Patient-Derived Dopaminergic Cell Model of Aromatic L-Amino Acid Decarboxylase Deficiency
Metabolomic Profiling in AADCd
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Professor Krystof
Poland/ USA
Update on AAV2-hAADC Gene Therapy Trials in Primary AADCd Paediatric Patients (Target Area VTA & SNpc)
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PTC Acquisition & AADCd GT Update

Measuring the Burden of Illness in AADC Deficiency

30MD: A Reliable Biomarker for AADC

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