3 Months on the Treatment & Starting PHASE 2!!!
Here is our official report on Damian's skills, abilities, and any other observable differences now that he's been on the experimental drug for THREE whole months!!
I just uploaded a YouTube video that shows really clearly just how much strength Damian has GAINED - I highly recommend watching!
I was so excited while editing this video and seeing what he can do now side-by-side with the footage from our 1-Month report! The progress, I truly believe, is indisputable!!
Each time I do this report, there are a few things that can't really be captured on video (circled in the graphic below). So I'm going to record my observations for those items here! I'll also include some high-level bulletpoints for each of the 4 categories.
Gross Motor Skills (Big Moves)
Biggest improvement is with his standing tolerance - he can stand up for so much longer and is doing so much better at pushing weight through his legs! I can almost not worry too much about holding his legs steady along with his torso - he almost holds his legs steady on his own!
His posture while sitting up is so much sturdier! I am now helping him practice using his arms to help him sit up - he can DO IT for about a second a time! I can't tell you how exciting this is!!
Damian can now pull himself along the ground about an INCH at a time. Not very often, and he takes very long breaks between each pull forward (about 5 minutes maybe?) but this is a VERY VERY big deal and SO encouraging!!!
Fine Motor Skills (Focused Moves)
Damian still really struggles with grabbing things and he doesn't like holding things, but the gained strength in his arms is so obvious when he does hold onto something and moves it. The YouTube video really shows this clearly!!
Speech & Communication
In the Month 1 Report, I said Damian really wasn't using many consonant noises. Well, they seem to be coming back!! Mostly "Gah" and "Dah" and "Nah," and he's not using them to form words, but he is babbling a lot - maybe a little more than he was before!
"Note what Damian Understands"
I just re-read what I wrote for this in the Month 1 Report, and to be honest, I was right when I said then that "this is probably going to be a tricky thing to monitor month-to-month." He still understands what he did then, and I'm not sure he understands more. He certainly understands a bunch of the words I say, like "daddy," "sandwich," "bottle," "ice cream," "yogurt," "food," "breakfast," "let's go," "bath," "all done," "look at mommy/daddy," "where's mommy/daddy?" and maybe others. I'm really hoping that Damian's communication is able to progress more!
"Note how Damian Communicates"
Damian still communicates through crying, laughing, smiling, and being excited. He does babble a lot, and sometimes he looks at me while he does it (as if I can understand him), but I don't think he is intentionally trying to say something specific. I have noticed him showing signs of frustration more often though, which could be a good thing because he might be trying to say something or wishing he could say something! Desire is the key to growth, right??
Feeding & Digestion
"Note nutrition & observations"
Oof. This one is tough. Damian really seems to be vomiting more frequently and more voluminously. I can almost certainly guarantee that that is due to Damian's liver and spleen. Neither of them seem to be getting any benefit from the brain treatment and instead are continuing to grow in size, squeezing Damian's stomach and squishing his lungs more and more. I CANNOT WAIT to start Enzyme Replacement Therapy (ERT): the 2nd experimental treatment we need for Damian, designed to shrink his liver and spleen down to normal size and, for all intents and purposes, will make them "healthy." ERT is already available through a formal clinical trial, THANK GOODNESS, and I've been working on getting Damian on this since November of 2021 (we put it on the back-burner when the brain treatment became within reach, since that was the priority and you can only start one experimental treatment at a time). But we are READY now and today we got some very very exciting news about the ERT that I will share tomorrow!!
No doubt because of the growing pressure on Damian's entire abdomen, Damian has been eating less over the past month. Instead of 2/3 of a PB&J, he's more often eating just half or sometimes less. He's drinking a lot of PediaSure, which is great, but I try to space it out and feed Damian solid food when I can because too much liquid makes him throw up even more easily for some reason. There are a lot of situations that can trigger a vomiting incident, but luckily Damian has not thrown up immediately after consuming his experimental treatment... that is, besides when we were starting Phase 2.
We are now in Phase 2!
Yes, that is my shirt soaking wet alongside both his shirt and pants inside one of the restrooms at Children's Hospital Los Angeles. Luckily the dark weathered gray color camouflaged pretty well the fact I was wearing a totally soaked shirt when we left the hospital (we still got looks though - not sure if they were noticing the wet clothes or the smell of vomit I wasn't able to get out). (Click here to watch a quick video of our day and that eventful incident at CHLA)
But let's focus on the positive - the HUGE positive was that we got to start Phase 2 of the experimental treatment study and even though Damian was not allowed to take another pill that day, Phase 2 meant that we didn't have to wait as long to take the next pill! In Phase 2 we moved from taking a pill every 3 days to taking a pill every other day! While I can't say I have seen a big difference since we increased the frequency of the dose a couple weeks ago, and most people say not to expect any drastic changes, I'm hopeful it will make at least a little bit of a difference! Time will tell!
I'm SO EXCITED to share our big news about ERT tomorrow!! And thank you so so much for continuing to share our story!! We are almost at "2 down, 1 to go" as far as treatments are concerned, and this last one is going to be our toughest battle yet. We have got to finish our campaign as quickly as possible because we don't just have to raise the money... the money has to be spent too. And then there is much more that has to happen afterwards to get the treatment from the lab to Damian's sweet little body.
I know we can do this. We did not come this far just to come this far.
We are doing this!!