Audrey's Gene Therapy Journey...
Q: How old was your child at the time of receiving GT and what is their
A: Audrey was 6 years old when she received Gene Therapy. She is now 7
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?
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.