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)?

 

A :  USA @ UCSF

 

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.

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