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Alex's Gene Therapy Journey...

Q :  How old was your child at the time of receiving GT and what is their

       current age?


A:  6 years old, he is now almost 8 years old


Q:  Before GT, would you have considered your child to be


       Mild (some physical function)

       Moderate (relying on medication for function)

       Severe (very limited physical function even with medication)


A:  Severe, Alex had very limited motor functions, could not roll, sit, or hold his head.


Q:  Which version of Gene Therapy did they receive – Taiwan/Japan (Target Area Putamen) or USA (Target Area VTA &                   SNpc)?




Q;  What was the recovery period after GT like and how long did it last?


A:  The physical recovery period (wound/fatigue) was about two weeks, the other effect of the GT lasts about 2 months.


Q:  Which symptoms of AADCd do you believe have improved the most by the treatment?


A:  OGC is gone, Congestion is mostly gone, reflux is gone, and he now has significant increase in motor skills and muscle tones.


Q:  Which medications after GT have you been able to stop taking?


A:  Specifically Dopamine Agonist. Still taking MAO b inhibitor and other supplements, as well as acid reflux meds (not for        reflux, but acid now impacts his GI greatly if not taking the meds)


Q:  Have you had to start taking any medication after GT?


A:  No new meds.


Q:  How has GT changed your child’s and family’s lives?


A:  His quality of life has increase dramatically as a lot of symptoms have disappeared. Family has less need to worry                about his health and day to day care. One issue arises is the mobility he gained is now becoming a potential safety  issue.


Q:  Would you recommend Gene Therapy to other families?


A:  Yes, although with risks, the quality of life improvement for the child is immeasurable and it offers hopes to the child as they continue to improve and develop after the GT.

Alex pic.jpg
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