Audrey's Gene Therapy Journey...

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

      current age?


A:  Audrey was 6 years old when she received Gene Therapy. She is now 7

      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:  Audrey was severe with limited physical function even with medication.


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


A:  USA (Target Area VTA & SNpc).

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


A:  The hospital stay was only 3 days but the first 3 months was tough for recovery. She is now at 10 months since Gene Therapy and is still progressing


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


A:  Audrey has not had an OCG for 9 months and can now participate in all activities at school.


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


A:  She has stopped taking all medications related to AADC


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


A:  No


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


A:  I have more head control and more energy throughout the day. This makes it a lot easier for all my helpers to help me with my daily routine.


Q:  Would you recommend Gene Therapy to other families?


A:  Yes. It is life changing experience.

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