I know this is a long post, but if you’ve got a few minutes, I hope you’ll read and continue your support of my family and others like ours. The more you know!
The Orphan Drug Act (ODA) passed in the US in 1983. Its purpose was to encourage drug companies to research and develop therapies for those with rare diseases. Why is it called “orphan”? Because, rare disease therapies generally lack sponsors to develop them. The market is small, research is expensive, and there is very little return on investment. There are over 30 million Americans suffering from over 7,000 rare conditions. Only 5% of those diseases have an approved treatment. Which means, the majority of those suffering have no treatment options at all. Taking your loved one home and being told to “make the best of the time they have left”, is a very real reality for many. Being granted an orphan drug designation by the #FDA, manufacturers and sponsors receive a 50% tax credit on qualified clinical testing, there are also several grants available. Which in turn helps recover development costs. According to the FDA, in the decade before the ODA was passed, only 10 treatments had been developed for rare diseases. That’s not a typo, TEN. This incentive, since inception, has helped bring nearly 500 new medications to patients. It has a proven track record of success.
Today, the House passed the Tax Cuts and Jobs Act. The Senate will vote after Thanksgiving. Included in this bill is the proposal to repeal or weaken the Orphan Drug Tax Credit. This is one of the only credits that is proven to save lives. I’m asking that you consider joining the #raredisease community in fighting to ensure it will not be weakened or repealed as part of the tax reform.
How can you help? Follow the link below. At the bottom of the form you will enter your address and zip code. A letter to your senators and representative will be filled out for you. The letter asks your representatives to oppose repeal or weakening of the Orphan Drug Tax Credit. All you need to do is enter your name, home address, email address and hit submit. It’s really that easy. I hope you’ll consider doing it.
As always, thank you so much for the support. xoxo
#themoreyouknow #wehaveHIhopes #hyperinsulinism #orphandrugs #taxreform
They say a picture is worth a thousand words, but looking at this picture, I could tell you THOUSANDS. These are lancets and test strips from our sharps container at home. These are all from glucose checks and not even half of what we’ve used over the last few months.
To me, these are hundreds of pokes on the sweetest baby toes you’ve ever seen. These are scared moments when his sugar has dropped too low. These are late nights and early mornings awoken by his crying. These are calibrations on his CGM. Testing how new foods affected his sugar. These are from moments when he was throwing up and we didn’t know how long we had before he’d become hypo, when we were checking every 30 minutes (or less), all while rushing in Houston traffic trying to get to the Texas Medical Center. But, mostly, thankfully, these were our reassurance that he was okay. This is what our lives have revolved around for the last nine months.
This is us checking for “Sugar Bugs” – no idea why we started calling it that but at some point, one of us said it and it stuck. How many sugar bugs are we going to find? Let’s catch lots and lots of them! Yay!!! We found 92! Seems kind of silly but we are trying to make it a positive and fun experience. He doesn’t cry anymore when the lancet pierces his skin or wake up when we poke him at night. His big toes are so callused that sometimes getting a small drop of blood is a challenge. I remember when we first started this journey, his poor toes became so beat up and turned red with bruises.
Now, here we are, just days away from the start of our “Cure Fast” at Texas Children’s. I am so anxious. We’ve slowly reduced his Diazoxide intake. He’s gone from 9.54mg/kg to 8.18mg/kg. So far he’s doing really well. He’s had a few numbers near 70 but for the most part, he’s hanging out in the 120 range. I pray that this is a good sign. I’m scared to be disappointed because no matter how many times I tell myself “Don’t get your hopes up”, they’re up. After all, I am a mom with HI Hopes. It’s who I am. I want this so bad for him, for us. The next two weeks, I suspect will be a roller coaster of emotions but I want to know. It’s the not knowing that has me feeling so crazy. If he has to live with HI longer, or even the rest of his life, we’re going to manage, I know that, but I really pray he doesn’t have to.
My hopes are that one day the words that come to mind when I look at that pile of lancets and strips are in past tense. That they become things that we used to have to do. Distant memories as a life free of hyperinsulinsm start making new ones in our lives. How sweet that life could be. I don’t know if that’s what’s in the future for us but I’m holding that dream close to my heart and never letting go.