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.

Speakers

A Welcoming Introduction to our 4th International AADCd Conference

An Overall Picture of the Structure-Function Relationship of AADC Variants Causing AADC Deficiency
Professor Marcello Coba
Modelling Human Disease with iPSC Derived Neurons
CSF Findings in a Carrier of AADC Deficiency/
Urinary Sulphatoxymelatonin
Metabolomic Profiling in AADCd
Natural History Study AADCd: A Retrospective Study
Update on MRI-guided midbrain Gene Therapy Trial in Children with AADCd
PTC Acquisition & AADCd GT Update
Next Generation Metabolic Screening for Elucidation of AADC deficiency and Related Neurometabolic Disorders
Conference Chair & Biochemical Introduction to AADCd
A Clinical Introduction to AADCd 
Generation of Patient-Derived Dopaminergic Cell Model of Aromatic L-Amino Acid Decarboxylase Deficiency
Successful Pregnancy in a Patient with L-Amino Acid Decarboxylase Deficiency: Therapeutic Management & Clinical Outcomes
Probiotics in DDC Mouse Model
Professor Krystof Bankiewicz USA
Update on AAV2-hAADC Gene Therapy Trials in Primary AADCd Paediatric Patients (Target Area VTA & SNpc)
Gene Therapy Case Studies in Japan

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

Measuring the Burden of Illness in AADC Deficiency

Next Generation Metabolic Screening for Elucidation of AADC deficiency and Related Neurometabolic Disorders

Dysonia Scale in Paediatric Neurotransmitter Disorders: A Pilot Study

Measuring the Burden of Illness in AADCd

Our Sponsors

30MD: A Reliable Biomarker for AADC

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The AADC Research Trust

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