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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To contact us please email via: enquiries@aadcresearch.org and we will endeavour to reply to you as soon as possible. We apologise in advance for any delays in our response, during this time. Our staff are on furlough and working from home. Please bear with us. Thank you for your patience.

 

The AADC Research Trust

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