AADCd Gene Therapy Trials: Japan / Taiwan

Target Area Putamen

Here is a collection of videos and articles relevant to the Gene Therapy from Japan / Taiwan which targets the Putamen area of the brain. 

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

Title: “Gene therapy improves motor and mental function of aromatic l-amino acid   decarboxylase deficiency”

Published: 22/1/19

Country: Japan GT

Title: “GT for AADC deficiency”

Published: 27/10/2017

Country: Taiwan GT

Title: “Gene Therapy in AADC”

Published: 12/1/2017

Country: Taiwan GT

Title: “AADC deficiency 2016”

Published: 10/6/2016

Country: Japanese GT

Here is an official publication which reports on 2 Gene Therapy patients in Taiwan, published in The Lancet 2017, entitled:

"Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial"

Please click on the image to read the full article..

 

 

 

Throughout the literature they refer to Patient 4 and Patient 7.

Please click on the relevant video to watch their development ...

Screenshot 2019-06-10 13.55.40.png

Patient 4

Patient 7

AADCd Gene Therapy Journeys...

 

Click on the links for more information on Daria's and Satoshi's Gene Therapy Journeys...

"It’s a great happiness not only to continue your children’s lives but to give them a chance to feel, to try and to enjoy a lot more than they can now. I see that GT is a big risk...but a huge piece of happiness you can present to your children is worth it! If you can just do it! Believe in magic!

Viktoriia (Daria's mum)

"I recommend GT. Satoshi can go out longer time on a wheelchair due to the mitigation of the respiratory problems He’s in a good mood and doesn’t cry. He can eat a small amount of food"

Akiko (Satoshi's mum)

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