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
An Overall Picture of the Structure-Function Relationship of AADC Variants Causing AADC Deficiency
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
Next Generation Metabolic Screening for Elucidation of AADC deficiency and Related Neurometabolic Disorders
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
Dr Riccardo Montioli
ITALY
Next Generation Metabolic Screening for Elucidation of AADC deficiency and Related Neurometabolic Disorders
Generation of Patient-Derived Dopaminergic Cell Model of Aromatic L-Amino Acid Decarboxylase Deficiency
Professor Krystof
Bankiewicz
Poland/ USA
Update on AAV2-hAADC Gene Therapy Trials in Primary AADCd Paediatric Patients (Target Area VTA & SNpc)
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30MD: A Reliable Biomarker for AADC
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