The past two months have been difficult for me. We have started the PTC 124 extension trial. We have waited for baited breath for the last two years to start this trial.
My son, who is more than willing to try any therapy if he thinks it will help, balked at the plan. I came home from work late one night to find my son in a state of total melt down. Yes, at 15 we have melt downs. For the past two months it has been a struggle to gag down the vanilla flavored drug. Given his age and weight his dosage is pretty high. His stomach has been upset; he has not been hungry and has experienced rapid weight loss. As I walked in the door he was trying to take his PTC and screamed at me "TAKE ME OUT OF THE TRIAL".
Those words stopped me in my tracks. I lost my mind, and slowly turned to my son and said, "FINE, I have wasted 10 years of hard work helping PPMD and the Duchenne community!" "Before we do anything, you really need to think about the end result will be!" My husband and I looked at each other and without speaking had the same question written on our faces. "What do we do now?" I turned to my son and said "OK, skip the dose tonight and go to bed, we will talk about it more tomorrow after we talk to the doctor."
My focus has always been on my son and on the bigger picture, whereas my husband's focus has been our son. I have always felt deep down that if this trial was really successful would change the face Duchenne and pave the way for other Duchenne trials. My biggest concern is messing up the protocol for the trial and putting future trials in jeopardy. Therefore, my constant mantra to my husband, as we try and figure out how to get my son to take this drug, have you checked with the doctor yet?
After checking with the doctor, we have stopped giving our son Juven and nearly all of his other supplements. The only medications he is taking are the ones prescribed and of course the Vitamin D and Calcium Supplements. Scary thought to us, as all the other stuff combined have worked and worked pretty well for the past 8 years. The up side is that our son is now able to tolerate taking this drug and is not experiencing the upset stomach and his appetite is returning to normal. So we will take it a day at a time and continue down the path we have begun
My hopes right now are:
The past two months and participating in this trial will all be worth it in the end.
All the trial participants will end up with a therapy for their sons.
By participating in this trial we will have helped pioneer future successful Duchenne trials.