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In 2019 we are focusing our Awareness Campaign on the impact that the pioneering Gene Therapy treatment has had on those AADCd children and their families, who have received it so far.

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Gene Therapy is complex brain surgery that has disease limiting and life changing results. This treatment has the potential to set the benchmark for many other neurometabolic diseases with a single gene defect and we are very proud to be a part of a journey that could drastically change the landscape in how we treat many RARE diseases.

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Currently there are 2 types of Gene Therapy being offered to AADCd patients:

 

1) AADCd Gene Therapy (Target Area Putamen) Japan/Taiwan

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2) AADCd Gene Therapy (Target Area VTA & SNpc) USA/Europe

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All week we will be delivering informative posts regarding both versions of Gene Therapy including statistics, videos, articles, patient insights, etc.

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Quote from an AADCd parent who's child has received Gene Therapy:

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How has Gene Therapy changed your child's and family's lives?

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"GT changed his life in so many ways. The quality of his life has improved so much. He can tolerate doing more fun activities than he used to. He can sleep through the night and is more awake and alert during the day. He acts happier in general. It has improved our familys life as well. We can do more together without worrying about OGCs. It is so much easier to carry him around now that he has more tone and is gaining weight. Plus, we have so much hope for what he will be able to do in the future".

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Would you recommend Gene Therapy to other families?

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"Absolutely! Even if having no OGCs was the only improvement he had gotten from the surgery, it would be totally worth it. That alone remarkedly improved his quality of life. He can sleep through the night and actually enjoy life during the day. He is so much happier. He has purposeful movements, which has to be such a neat experience for someone who could never do that. He now has such a better quality of life, which to us was worth the risks of surgery. We would do it all over again if we had to".

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 #CureAADC.