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.
