When I placed a call on Monday to the infusion doctor, I found out that a committee was meeting on Tuesday to discuss the Brineura treatment. I didn’t have the chance to post that on here beforehand but yesterday I received a call from our genetic counselor with a summary of the meeting. I want to update you on what we know now.
At this meeting were several doctors, some hospital staff, and 2 reps from BioMarin—10 people total. To start, let me share something that was a blessing to my heart. Our genetic counselor said she has worked at OU Children’s for 3 years and has never seen 10 people come together to discuss just one family, more specifically one little girl. I, too, have been overwhelmed with that thought lately. I’m thankful for doctors, nurses, etc. that show care and compassion. In my heart, I want to say we aren’t through seeing things we haven’t seen before. Whether the outcome is what we desire or not, we will see things done that only God can do.
First of all, we know that BioMarin will not release the drug into the market until June 5 or 6. That’s just a little over 3 weeks away though. In the meantime, we hope that some things can be settled. The treatment has been submitted to insurance. We hope to make some headway with that in the next 3 weeks. Of course, because it is so new, there will be hold ups. Based on the information we find after a couple weeks of the insurance process, we will decide if she has the surgery before June 5. It would be nice to have the surgery and give recovery time so that she will be ready to receive the infusion when it is released. We can all pray that way!
We also received the name and number of the local BioMarin rep. Now, I can call her with questions or check on progress. That is great! She will also get us information on a program that BioMarin has called Rare Connections. This is how BioMarin states the mission for RareConnections. “BioMarin RareConnections will work with patients, physicians, and insurance providers to make sure that patients receive the treatment they need and ongoing support, no matter their situation. An experienced BioMarin RareConnections team will help guide patients and caregivers around barriers to treatment, financial hurdles, and life changes that may come between them and the care they need. This includes help with obtaining coverage, minimizing out-of-pocket expenses, and finding alternative financial assistance for treatment.” It will be nice to have someone knowledgeable to help guide us through treatment and all that’s involved. We will also be able to enter the registry BioMarin is creating for NCL patients.
Two items of good news: 1. The infusion doctor at OU Children’s will do the infusions at first and she already knows how to do these types of infusions. No one will have to be trained for us to get started. 2. The pharmacist present at the meeting says OU Children’s already has all the equipment needed to do the infusions so no purchase of new equipment needed. I think those are direct answers to prayer. Both of those will save time and money. Praise the Lord!
Joyanna had a couple of better days at the beginning of this week. She was moving around a little more. Yesterday, she seemed very tired and today, she was just not having a good day to begin with. We can see that her seizure level has increased. We’ve been at a hard place because we thought we really couldn’t raise any of the current medicines she was on and didn’t want to start her on a new one right now with the treatment so close at hand. We did find out yesterday that we can slightly raise the medicine to which she responds best. Please pray this slight change will give her relief from the seizures.
Philip has had pretty good days over all. He was able to start back on Zyrtec and has responded well. Yesterday, he was a little more congested and had a few more seizures at school but he slept well last night and seemed better this morning. He has made it to all 5 days of school this week. His last day of school is May 30. We already have the ball rolling to start home health care for him this summer. The Lord is making paths clear there so we are thankful. Hopefully, a family friend from our church will be able to be his nurse once insurance is processed and the actual health care started.
Ethan is back on the potty training wagon but it hasn’t been without its bumps. I’m quite certain I will never speak up to give the potty training advice when a younger mother has questions. I’ll gladly defer to someone else. In fact, if anyone wants to host a 3-year-old for a couple of months and bring him back to me potty-trained, I would consider the offer. Ha Ha! I guess we would miss our little “entertainer” if he was gone that long.
Well, the school year is over at Heartland Baptist Bible College. (My husband is the Dean of Men there.) The summer stretches out before us now. It won’t have long to stretch. It seems to go so fast these days. We hope to enjoy a slightly slower pace for a couple of months.
Once again, thanks for praying for us. We hope that you can see God working and see that your prayers have direct ties to His work in our lives.
This is one of our favorite songs to listen to for years now as we navigate this journey.
He Who Holds Us In His Hands
When God says no to you, it’s such a blow to you,
When all your hopes and dreams unravel at the seams,
When God says wait to you, and God seems late to you,
It’s hard to rest within His will, it’s hard to trust and just be still,
It’s hard to simply wait until it’s all made plain.
Chorus
He who holds us in His hands has no problems only plans.
He who has control of all sees the smallest sparrow fall.
He who works where no one knows does not sleep as we suppose,
But works in all things, both great and small things,
For His own glory and our best good.
When God works silently in ways we cannot see,
When troubles multiply so that we ache inside,
When God does not seem kind, and hope is hard to find,
It’s hard to rest within His will, it’s hard to trust and just be still,
It’s hard to simply wait until it’s all made plain.
He who holds us in His hands has no problems only plans.
He who has control of all sees the smallest sparrow fall.
He who works where no one knows does not sleep as we suppose,
But works in all things, both great and small things,
For His own glory and our best good.
For His own glory and our best good!
(Composer: Bryan Jeffery Leech)