Kacper's Gene Therapy Journey...

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

      current age?

 

A:  Kacper was 12 years and 11 months old when he started GT. Now he is

     13 years and 1½ months 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:  Before GT my son was diagnosed as: Severe (very 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:  Kacper was given the GT version USA.

 

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

 

A:  After GT my son left hospital after 3 days in a good stable state. From 5 to 7 weeks after GT he had the most severe                        dyskinesia and he had big problems with sleep. At present the dyskinesia has continued , however at a lower level.

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

 

A:  As a result of treatment up to now things which have most improved are:

 

  • No more nasal congestion, as if it had a cold - it has completely stopped.

  • Up to today no OGC for two months.

  • Physically: raises his head when on his stomach, can remove his head from the back of the wheelchair and controls it, he can control over his head in a seated position without support, his head does not fly on to his back, whilst moving from lying position to sitting position he can control his head, he moves his arms/hands better and can connect them, he makes lots of sounds, you can hear its voice, he laughs loudly like a healthy child, this laugh has the same intonation as his voice, it is different than before the therapy.

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

 

A:  After GT, my son stopped taking pramipexol and tranylcypromine.

 

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

 

A:  Up till now, Kacper has not had to take any drugs after GT.

 

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

 

A:  It is only 2½ months after GT, however when I see the progress my son has made in his movement, there is probably no                greater joy for everyone involved, especially for the mother. Kacper is very happy, I can see the joy in his eyes. This is amazing.      It is impossible to describe my happiness in words.

 

Q:  Would you recommend Gene Therapy to other families?

 

A:  I would highly recommend GT. Children have a chance for a new, a better life. I would like for all children with this disease to        be able to receive GT treatment and live as close to a normal life as possible, so they are not chained to strollers or beds. I              thank God that my son has received GT and I pray for everyone who made it possible.

Gene Therapy : Target Area Substantia Nigra Pars Compacta & Ventral Tegmental

(based on GT approach developed under NHI grant, approved by the FDA, 2 US IRB's and constitute experimental treatment to save lives of ADAC deficient children).

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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.

 

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