Tuesday, May 12, 2015

Great News from the FDA!

This afternoon, the FDA Advisory Panel recommended approval of the latest CF corrector drug, formerly VX-809/lumicaftor combined with Kalydeco, now known by it's official brand name, Orkambi! The final vote was 12 yes, 1 no. This is not the official and final approval, as the final word is expected to come on July 5th. But I learned today that the FDA uses the panel's recommendations to make their final decision, and they typically go with what the panel recommends. So while we still have to wait a couple months to find out if the portion of the CF population that carry 2 copies of DF508 and are ages 12 and older will soon have their magic pills in hand, we're significantly closer than we were before, and the remaining wait isn't much longer.

Orkambi's results weren't what we were hoping for, and during the studies it didn't show the improvements that Kalydeco had on patients with the G551 mutation. But there is enough improvement to slow the progression of the disease, which means it will buy more time for the double DF508 population, and Vertex can keep the momentum going on other corrector drugs like VX-661. And once this is approved, they can start the studies for the ages 6-12 group, and then the 2-6 group.

It's a bittersweet day, and July 5th will be bittersweet as well. If you've followed us for a while, you know I've talked about these drugs before, and have also mentioned that Judith only has 1 copy of DF508. Orkambi won't help Judith. The recommendation of approval, and (hopefully!) the final approval of Orkambi is another reminder that we're still waiting for our turn, waiting for our magic pills. All while we have to do whatever we can to keep her as healthy as possible as the disease slowly progresses. Our turn will come. I don't know when, but we'll get there. The waiting game absolutely stinks, but someday we'll join the ranks of CF patients who have a corrector drug that will benefit them.

At the same time, I'm rejoicing for my many friends whose loved ones will benefit from Orkambi, and any future correctors that are better! A chunk of my friends have children who are under the age of 12, so they won't have full access right away (but might be able to get it off-label), but it may not take long until the studies in the other 2 age groups are completed, and their children can start taking Orkambi.

I'd be lying if I said I wasn't tense today. I knew the panel recommendations were coming, but after a link to a live blog was posted in a group I participate in, I knew I'd be glued to my computer when I could. I literally let out a huge sigh of relief seeing the final vote results! Now to wait for the FDA's final decision...

Thursday, April 30, 2015

Quarterly Clinic Visit

In true spring fashion, Judith had (more like has, because she's still not over it) a nasty cold for this clinic visit. Naturally, I was sick as well, although I was way over the hump and not contagious at that point, even though my voice still sounds horrible. In some respects, I hate taking her to clinic when she's sick with a respiratory bug, because that only means the potential for extra germs to end up on surfaces, but at the same time it works out well because they can hear what she sounds like in person, and tailor her treatment plan accordingly.

If you've been keeping track, this is respiratory infection #4 for her over the last 6 or so months. This is also exacerbation #3, with the 3rd round of steroids. And we learned something new: steroids make my child go absolutely bat crap crazy. The behaviors that accompany her ADHD are exacerbated by the steroids, and she literally becomes a wild woman. I told Dr. G. that we absolutely will NOT use that particular steroid again unless it's a dire situation or the absolute last thing available. He did say that there are some other brands we could try and see how she reacts, but it could be the steroids in general that cause this. I have to say that her behavior this time was especially difficult, because I was trying to fight off bronchitis at the same time, and John's schedule was also insane so most nights of the week I had to fly solo. It was the perfect storm of events, and it was especially trying. Nevertheless, her behavior was that out of control within 15-30 minutes of taking her steroid dose that I don't think it would have been any less exasperating had I been feeling well.

Judith got to try something new at clinic this time! Our clinic has patients start blowing PFTs at age 5, and starts practicing around age 4.5. Judith's not quite 4.5, but when the nurses asked about her age, I pushed a bit and requested starting practice now, because if we can get her started and into the routine earlier, she may not be as nervous or scared about the process and will likely be more willing to cooperate. So the RT came in with a party blower, and we practiced with that for a few minutes! I'm going to be making a trip to the dollar store to get a pack so we can practice at home, and at her next visit she'll get to try it out with the machine.

Judith grew again, and is now 40.25" tall! I knew she was growing again, because all of her 3T pants, while still a perfect fit in the waist, are too short and make her look like she's wading through shallow water. The size 4T/4 pants I have are still slightly long, but are definitely fitting better than the older stuff. Weight gain was not impressive at all, because she barely gained anything since her last appointment. On Monday, she weighed in at 39 lbs 9 oz. So that's an increase of just a few ounces. We're waiting it out to see if she can gain more weight by her next visit, particularly since she's been sick and had back to back infections.

Everything else seemed ok. There is some mucus in her lungs, but she has been showing signs of clearing it out via treatments, and Dr. G. didn't say that he heard anything extra concerning. We're waiting for the results from her cultures, and are hoping that nothing new cropped up that could be causing her wet cough (in other words, we're hoping that it's just the respiratory virus and not something like pseudomonas).

Wednesday, April 8, 2015

VeggieTales: Noah's Ark Review

*** I am not being compensated in any way for this review. Please note that it is purely my opinion, and that of my highly opinionated 4 year old. ***

I'm stepping outside of my box a bit, because I felt compelled to write a review here about the new VeggieTales movie that was released a few weeks ago.  I think it's because VeggieTales are a staple in our house, and the shows/movies are frequently Judith's go-to choices during treatment times.

This past fall, some big changes happened to a huge franchise in the Christian community. For 20+ years, Big Idea produced many videos in the VeggieTales series, promoting Christian values and retelling Biblical stories in their own, unique way. But as they were developing a new series for Netflix, a total overhaul of the beloved characters occurred; the design was drastically changed, and many of the voices were also changed.

Did you ever hear the old joke about Lutherans?

How many Lutherans does it take to change a lightbulb?
Change?  What's change?
 ::cue laugh track::

I'm a lifelong Lutheran who will readily admit that I hate change. I'm not totally resistant to it, and at times I even embrace it, but that doesn't mean I won't complain at times. The changes to my beloved Veggies really irritated me at first. Like, REALLY irritated me. Eventually I started to accept the redesign in small ways, but never fully accepted it. I wanted to try and accept it as quickly as I did with other redesigns (like the redesign of My Little Pony, for example), but it wasn't happening. The other kicker that prevents me from fully accepting it at this point: the voicing changes. Some of the major characters are the same, but many are not, and it's more than noticeable and actually upsets Judith.

You may be wondering what all of that has to do with the latest VeggieTales movie, Noah's Ark. It actually has a lot to do with it, because this movie uses the new design and the new voicings.  I'm going to break my review down into a few categories, and then give my overall rating.

The story line is classic VeggieTales. Obviously, since it's the story of Noah's ark, it's based off the account from the book of Genesis. There is a unique Veggie twist to the story, such as the ark being a giant orange slice. It's similar to other shows/movies in the franchise that pull the story from the Bible, like Joshua and the battle of Jericho, Jonah and the big fish, etc.

One big difference that I picked up after the first viewing, and now with subsequent viewings, is the omission of some of the "punny" humor and subtle jokes that kids wouldn't necessarily pick up on, but the adults watching with them would get. There is a little bit here and there, but not nearly as present as it was in the past. I miss that. I'm not overly bothered by most children's shows, and can certainly tolerate watching them with Judith, but when you have something like VeggieTales that stands out with adding some humor for the teens and adults watching and all of a sudden it's gone, a fan is definitely going to notice.

Otherwise, the story was generally well done.

This part is a hot topic around the internet. Like I stated earlier, I accept the design in some ways, but haven't fully embraced it. I understand why they did it, but at the same time I wish they wouldn't have messed with a good thing. The new design is certainly more streamlined and modern, but it's the small things that are irritating. Things like Madame Blueberry; I mean, was it really necessary to put bags under her eyes? The poor blueberry now looks like she's constantly sleep deprived. Junior Asparagus is now missing his trademark hat, something that Judith has commented on ("Mommy, where's Junior's hat?"), and to be extra frank, she now has to think about who he is because he looks like a run of the mill asparagus and doesn't have his unique, prominent identifier. And the French Peas? The overexaggerated mustaches make them look bizarre at best.

My biggest sticking point is the eyes. It's not necessarily the color that was added. Want to add color? No biggie. It's the fact that now they don't have nearly as much life as the previous designs did, and if I'm completely honest, they're kind of creepy. They remind me of similar eye designs on children's cartoon characters like Daniel Tiger. It's that vacant, almost piercing stare that does it. It's one of the design features that I feel has turned the franchise into another "dime a dozen" cartoon, which has cheapened the franchise. Yes, they're still kind of unique because hey, they're produce, after all. It's just disappointing that they're not as unique as they were with the original designs, and don't stand out quite as much.

Judith noticed the difference with the eye design rather quickly. It didn't take long for her to declare that she did not like their eyes, and she said she likes the "old ones better."

This is another sticking point with me, and at this point it's probably the biggest deal breaker.  Judith is not thrilled with it either, particularly with the change in Junior's and Petunia Rhubarb's voices. It was the one thing causing me to hesitate when I was deciding what VeggieTales DVDs to get to put in her Easter basket, and I wasn't having any luck with my searches. Scuttlebutt online was that the voices would be the same as they previously were, with the re-voicing only happening for the Netflix series. Now I know that this is not the case.

Some of the characters did retain their original voicing, namely those voiced by Phil Vischer and Mike Nawrocki. So there is some consistency with the older shows/movies. It's the characters that were re-voiced that's bothersome and causing Judith to ask questions.

One stand out, though, is Wayne Brady. I'm a big Wayne Brady fan, and when I saw that he was voicing Shem, that was the one thing that helped me to decide to pre-order the DVD and give it a whirl. Wayne Brady really did do a fantastic job, and I kind of wish they would've tried to feature him in a previous show/movie.

Summary of Thoughts
Overall, the story is nice and has the familiar feel of a Biblical story with a VeggieTales twist, but the lack of puns and other humor that was so prevalent in the past made Noah's Ark fall a bit flat. It didn't jump out and wow me like the shows/movies of the past have done. Combine that with the redesigns, and I'm not totally sure that I will be spending money on buying the actual DVDs to add to our collection, at least not right away. For now, new releases are going to be viewed on Netflix or another streaming site that carries it first, because the redesign is not holding Judith's attention like the old designs were. In the past year or so, I knew I could buy any VeggieTales DVD for her, and it would be a hit, but now? Not so much.

My Rating
On a scale of 1-5 stars, 1 being the worst and 5 being the best, I give Noah's Ark 2.5 out of 5 stars.

Monday, April 6, 2015

Easter 2015

We want to wish Christians everywhere a very happy and blessed Easter!  Christ is risen, He is risen indeed!  Alleluia!

Tuesday, March 10, 2015

The Neverending GI Story

This is another post about poo.  I want to give fair, advanced warning for those that may not care to continue onward.

Still reading?  Cool.

As you may remember, we're still working on potty training Miss Sassypants.  She was doing really well with pooping, and we knew the miralax was working and keeping her cleaned out.  Recently, though, she started exhibiting some behavioral issues in regards to elimination, and started holding it in instead of letting it go.  We all knew she had to go, including Judith, but she would try and hold it in until the next day and was miserable as a result.

Now we're having the additional problem of increased mucus production because she appears to be brewing yet another respiratory infection.  All of that mucus her vest shakes out of her lungs has to go somewhere, and there are basically 2 options: spit it out, which is something we're going to start working on so she can then start learning how to huff cough; or swallow it, which is typically what young kids do until they learn to spit it out.  From there, it has to get out somehow.  Sometimes if it's really bad and upsetting her stomach, she'll vomit it out.  Most of the time, though, the only exit point is through her intestines, and that leads to some really mucusy poo.  The other problem with this?  If it doesn't exit, it hangs out in her intestines and blocks things up/slows things down.

Over the weekend, we knew she had to go, but it just wouldn't work it's way out.  I started to panic Monday morning because we were starting day 2 with no results, and I was concerned she was backed up.  1 phone call to clinic later, and we got the green light to start an at home clean out with multiple doses of miralax.

We loaded her up, and she kept trying to go, but was saying it was stuck, and eventually started saying that it hurt.  By 7:30, she started screaming and straining.  This has happened a few times in the past, most notably when she's had the 2 significant blockages that required inpatient clean outs, so I figured we could be in for a rough hour or 2 while she tried to pass whatever was sitting there.  But once she did go, the screaming didn't stop.  This wasn't the typical backed up poo, but we couldn't find evidence of anything else happening, like a rectal prolapse.

I knew I needed to call clinic and ask them about other possibilities, and the first thing they thought of based off my descriptions was rectal prolapse.  So my line of thought wasn't too far off base, even though we didn't see anything.  It's very possible that it could have happened, but retracted quickly once she relaxed.  We're certainly going to have to keep an eye on it, but it sucks that this is even remotely in the realm of possibility for her.  It's something that I've been concerned about for a long time since she's always had to strain to go, but if it is starting to happen and as long as it's retracting on it's own, it shouldn't be as problematic as it would be if it weren't able to retract.

Today has been a much better day for her, and I'm hoping that the higher doses of miralax will keep things moving and help push back at the mucus that could be building up in there.

Monday, February 23, 2015

I Am Not A Supermom

When you have a child with special needs, you hear some interesting things come out of the mouths of other people.  Sometimes it's unsolicited advice, which every parent gets; sometimes it's a comment or statement that is completely asinine and rude; sometimes it's advice that is well intended, but can't be applied to your situation, and can sometimes lead to hurt feelings; sometimes it's a statement of awe or wonder.

"I don't know how you do it!"

"You must be a supermom to handle everything!"

And the above statements are sometimes followed with "I know I certainly couldn't do it!"

Have I ever made these statements?  Absolutely.  These days I often say it jokingly with my friends who have 2 or more kids.  Personally, I don't see anything harmful in the first and last statements, either as a stand alone statement or combined.  Even the middle statement is relatively harmless compared to other things that have been said to me in the past.  But for some reason, the whole "supermom" sentiment bugs me a bit.

Dear readers, I want to say that I am not a Supermom.  I'm a mom just like the countless other moms in the world.  I make mistakes, just like every other mom in this world.  Sometimes a treatment is missed.  Sometimes housework sits and waits for a while because there just aren't enough hours in the day to do enough treatments while on sick plan.  Sometimes I'm constantly getting up and down to deal with poop and mucus, and sometimes I'm getting snot or the latest haul from the nose mine wiped on my shirt because she "forgot" to go get a tissue.

How do I do it?  I just do.  Which is exactly what every single other parent in the world does with their child, special needs or no special needs.  I don't possess any magical powers that help me soldier through.  I know that because of Judith's special needs, I'm required to wear more hats than a parent who has a typical, healthy child.  Some days are hard.  Really hard.  And those are the days when I may wish I had some super powers, or at the very least had some extra sets of arms to help with a few more things.  But at the end of the day, I know this is our reality, and I do the best I can with what I have.

The first year with a new baby can be overwhelming for anyone, but when you throw special needs into the mix, it really can make it that much more challenging and overwhelming.  You adjust and adapt, though.  Routines are established.  When you think you have this little human figured out, they go and throw you for a loop and you have to go back to square one and figure things out again.  That seems to be the typical pattern, and even with the special needs thrown in, we went through that entire process.  Some of the differences were trying to figure out treatment processes, getting ourselves organized in the best way possible, and learning to cope with the emotional aspects of this disease (which can still get you at any given time, but you get better with it not hitting as frequently).

I have to say that even though I was pretty organized in the past, I became almost fanatical about it shortly after she was born, and am still an enthusiast.  That is one of those things that almost has to happen until you can do the new routine in your sleep.  Doing that doesn't require anything special; you figure out what's going to work best for you so you don't go crazy trying to track and remember everything.

I am not a Supermom.  I'm just a mom who has a different reality, doing the best I can to give my child as normal of a childhood as possible, all while working through and with her challenges.

Friday, February 6, 2015

T-Shirt Fundraiser!

We're always looking for people to join our Great Strides team, and even if you can't be at the walk in person, you can sign up to be a virtual walker!  If you'd like to do that, you can sign up here: Judith's Crusaders

If you can't walk, we have a new option for you to show your support for Judith's Crusaders!  You can buy one of our snazzy team tees, and let everyone know that you're joining the fight for a cure!

We have 75 shirts available, and our fundraiser will run through the end of the month.  Shirts will be delivered mid-March.  They are $20 each, and are available in many different sizes.  Order your shirt here: http://www.booster.com/judithscrusaders2015

Of course, if you don't want a shirt, you can always donate directly to our team using the link provided above to sign up to walk with the team.