Satoshi's Gene Therapy Journey...
A: How old was your child at the time of receiving GT and what is their
Q: At the time of receiving GT, Satoshi(my son) was 19 years old and is now
22 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: Satoshi was bedridden, tube feeding, and had severe respiratory problems and epilepsy. I considered that my son was severe one.
Q: Which version of Gene Therapy did they receive – Taiwan/Japan (Target Area Putamen) or USA (Target Area VTA & SNpc)?
A: Satoshi received Taiwan/ Japan version (Putamen).
Q: What was the recovery period after GT like and how long did it last?
A: Satoshi recovered from damaged by the surgery in a few days. He had the involuntary movements (as a side effect) for 3 to 6 months after GT.
Q: Which symptoms of AADCd do you believe have improved the most by the treatment?
A: Respiratory problems
Q: Which medications after GT have you been able to stop taking?
Q: Have you had to start taking any medication after GT?
Q: How has GT changed your child’s and family’s lives?
A: He 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.
Q: Would you recommend Gene Therapy to other families?
A: I recommend GT. Satoshi got GT at 19. Although he could not get significant recovery of his motor function, he got a mitigation of the respiratory problems. I think if he had not got GT, he would have had a tracheotomy. It was a grave thing for us to avoid the tracheotomy As for negative side, he had a progression of a scoliosis after GT. Satoshi now control the scoliosis with the periodic PT and a trunk supporting apparatus.