Satoshi's Gene Therapy Journey...

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

      current age?


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?


A:  Rotigotine


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


A:  Alfacalcidol


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.

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