Logan's Gene Therapy Journey...
A: How old was your child at the time of receiving GT and what is their
Q: Logan was 4 years old when he received GT and is still 4 years old, he
will turn 5 in July.
A: 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: Logan was definitely severe. He had no head control, very minimal purposeful movements and extremely low tone. Medication did not seem to make any difference.
Q: Which version of Gene Therapy did they receive – Taiwan/Japan (Target Area Putamen) or USA (Target Area VTA & SNpc)?
A: Logan received the USA version in San Francisco.
Q: What was the recovery period after GT like and how long did it last?
A: The recovery period for Logan was really short considering the fact that its brain surgery. He only spent about 2 days in the ICU and then we were able to go back to our room at the Family House. Once we were out of the hospital, he didn’t need any pain medication other than some Tylenol and he seemed pretty comfortable. The first day or two was the worst of it, Logan had a lot of swelling around his face and seemed uncomfortable. After that, he seemed comfortable.
Q: Which symptoms of AADCd do you believe have improved the most by the treatment?
A: Logan has not had an OGC since about 2 weeks post surgery. His sleeping issues have improved dramatically, he is able to sleep through the night without waking. He has some head control now and he has improved tone throughout his body. He cries so much less.
Q: Which medications after GT have you been able to stop taking?
A: Logan was able to stop taking his evening dose of selegline. He was not on a dopamine aganoist before surgery.
Q: Have you had to start taking any medication after GT?
Q: How has GT changed your child’s and family’s lives?
A: GT changed Logan’s life in so many ways. The quality of his life has improved so much. He can tolerate doing more fun activities than he used to. He can sleep through the night and is more awake and alert during the day. He acts happier in general. It has improved our family’s life as well. We can do more together without worrying about OGCs. It is so much easier to carry him around now that he has more tone and is gaining weight. Plus, we have so much hope for what he will be able to do in the future.
Q: Would you recommend Gene Therapy to other families?
A: Absolutely! Even if having no OGCs was the only improvement he had gotten from the surgery, it would be totally worth it. That alone remarkedly improved his quality of life. He can sleep through the night and actually enjoy life during the day. He is so much happier. He has purposeful movements, which has to be such a neat experience for someone who could never do that. He now has such a better quality of life, which to us was worth the risks of surgery. We would do it all over again if we had to.