Gene Therapy:

USA / Poland

Target Area Substantia Nigra Pars Compacta & Ventral Tegmental 

Here is a collection of video's and articles relevant to the Gene Therapy from USA / Poland which targets the Substantia Nigra Pars Compacta & Ventral Tegmental area of the brain.

They also document the progress made by some of the AADCd children that have received this form of Gene Therapy.

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Title: "Experimental Therapy Gives Children With Genetic Disorders A New Chance At Life"

Published: 19/03/2019 KPIX5 News

Country: USA

AADC World Conference 2018

Professor Krystof Bankiewicz & Dr Toni Pearson presentation:

Title: “AADC Krystof Bankiewicz & Toni Pearson - Phase 1 AAV2-hAADC Clinical Trial”

Published 13/12/2018

Country: USA / Poland

AADC World Conference 2014

Dr Toni Pearson presentation

Title: “AADC Gene Therapy Trial & Natural History Study”

Published 23/12/2014

Country: USA / Poland

Title: "Amazing advances in the treatment of childhood Parkinson's"

Published: 17/02/2019

Country: USA / Poland

AADC World Conference 2014

Professor Krystof Bankiewicz presentation

Title: “Overview of AADC Deficiency - AAV2-hAADC Gene Therapy Trials”

Published 23/12/2014

Country: USA / Poland

Title: “Neuroterapi Center - a new gene therapy for the treatment of brain diseases” (Bankiewicz introduction to his polish clinic/lab) 

Published: 4/3/2016

Country: Poland

Active Gene Therapy Trial Information:
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A Single-Stage, Adaptive, Open-label Dose Escalation Safety and Efficacy Study of AADC deficiency in Paediatric Patients(AADC)

Sponsor : Krystof Bankiewicz

Collaborator: National Institutes of Health (NIH)

Information provided by (Responsible Party):

Professor Krystof Bankiewicz, University of California, San Francisco

This trial is ACTIVE and RECRUITING.

If you are interested in registering for this trial please click on the image to find out more information. The contact email addresses are at the bottom of the link page.

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