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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