News, Stories, Research Grants, and Announcements!

MPSIIFund News

Sleep Study & a Thank You

Dr. Moxham (Trey's ENT) and Trey. Winter 2006/2007.


Last night was a quiet and fun (and a bit lonely) night. Trey had a sleep study at BCCH and Ryan took him (Ave is still breastfeeding and only one parent can accompany a child). From 6pm-7:15am, it was just me and Avery. Wow it was quiet. And pretty great, spending some time with just me and Ave, since that very rarely happens, and never for that length of time. It was however, lonely, when I went to climb into bed and both my husband and firstborn were missing from our huge bed!!
Apparently the sleep study was a bit of a gong show. Trey was hooked up all over the place, so wasn’t very comfortable, and he kept asking for Ave and I. Ryan went to work late today with less than 5 hours of sleep. Poor guy.
On a note of thanks, last week we received a cheque from the MPS Society Bone Marrow Transplant Family Assistance Program for $1,500, which helped alleviate the costs associated with our trip to North Carolina. Fundraising and donations are what allows the Society to have a program like this which helps families in times of need. Thank you to the Society!
And on the ERT front, things are moving along. Still no dates, but all arrangements have been made and all we’re waiting for now is for Shire to send the drug up to Canada. In the meantime I have been researching infusion rates (which is not as straightforward as I thought) and reactions (which are not all as benign as I thought).
And then tomorrow we have our pre-op appointment with the anesthesiologist, and Wednesday Trey has his VAD surgery. Compared to other surgeries, this one isn’t a very big deal, but it is Trey’s biggest surgery to date, and letting go of your child as he falls asleep into the hands of a stranger who is going to cut your child open is not easy. However, I’m not really worried because Trey is so strong and healthy and great! And on top of it, the surgery is a good thing. He’s getting it not because anything is wrong, but because everything is right! He’s about to start ERT!!!!!!!!!!!!!

Sniff, sniff…

Trey, on Father's Day in June 2004, asleep in the swing chair.


Ryan’s cousin (she has 3 kids of her own) sent this to me this morning. She saw it on a forum she participates in and I want to share it with you…
“My friend is expecting her first child. People keep asking what she wants. She smiles demurely, shakes her head and gives the answer mothers have given throughout the pages of time. She says it doesn’t matter whether it’s a boy or a girl. She just wants it to have ten fingers and ten toes.
Of course, that’s what she says. That’s what mothers have always said. Mothers lie. Truth be told, every mother wants a whole lot more. Every mother wants a perfectly healthy baby with a round head, rosebud lips, button nose, beautiful eyes, satin skin and straight feet. Every mother wants a baby so gorgeous that people will pity the Gerber baby for being flat-out ugly. Every mother wants a baby that will roll over, sit up and take those first steps right on schedule (according to the baby development chart on page 57, column two). Every mother wants a baby that can see, hear, run, jump and fire neurons by the billions. She wants a kid that can smack the ball out of the park and do toe points that are the envy of the entire ballet class.
Call it greed if you want, but we mothers want what we want. Some mothers get babies with something more. Some mothers get babies with conditions they can’t pronounce, a spine that didn’t fuse, a missing chromosome, a palette that didn’t close or a tiny crooked foot or two. Most of those mothers can remember the time, the place, the shoes they were wearing and the color of the walls in the small, suffocating room where the doctor uttered the words that took their breath away. It felt like recess in the fourth grade when you didn’t see the kick ball coming and it knocked the wind clean out of you.
Some mothers leave the hospital with a healthy bundle, then, months, even years later, take him in for a routine visit, or schedule her for a well check, and crash head first into a brick wall as they bear the brunt of devastating news. It can’t be possible! That doesn’t run in our family. Can this really be happening in our lifetime?
I am a woman who watches the Olympics for the sheer thrill of seeing finely sculpted bodies. It’s not a lust thing; it’s a wondrous thing. The athletes appear as specimens without flaw – rippling muscles with nary an ounce of flab or fat, virtual powerhouses of strength with lungs and limbs working in perfect harmony. Then the athlete walks over to a tote bag, rustles through the contents and pulls out an inhaler. As I’ve told my own kids, be it on the way to physical therapy after a third knee surgery, or on a trip home from an echo cardiogram, there’s no such thing as a perfect body. Every body will bear something at some time or another. Maybe the affliction will be apparent to curious eyes, or maybe it will be unseen, quietly treated with trips to the doctor, medication or surgery.
The health problems our children have experienced have been minimal and manageable, so I watch with keen interest and great admiration the mothers of children with serious disabilities, and wonder how they do it. Frankly, sometimes you mothers scare me. How you lift that child in and out of a wheelchair 20 times a day. How you monitor tests, track medications, regulate diet and serve as the gatekeeper to a hundred specialists yammering in your ear.
I wonder how you endure the clichés and the platitudes, well- intentioned souls explaining how God is at work when you’ve occasionally questioned if God is on strike. I even wonder how you endure schmaltzy pieces like this one — saluting you, painting you as hero and saint, when you know you’re ordinary. You snap, you bark, you bite. You didn’t volunteer for this, you didn’t jump up and down in the motherhood line yelling, “Choose me, God. Choose me! I’ve got what it takes.” You’re a woman who doesn’t have time to step back and put things in perspective, so, please, let me do it for you.
From where I sit, you’re way ahead of the pack. You’ve developed the strength of a draft horse while holding onto the delicacy of a daffodil. You have a heart that melts like chocolate in a glove box in July, carefully counter-balanced against the stubbornness of an Ozark mule. You can be warm and tender one minute, and when circumstances require, intense and aggressive the next. You are the mother, advocate and protector of a child with a disability. You’re a neighbor, a friend, a stranger I pass at the mall. You’re the woman I sit next to at church, my cousin and my sister-in-law. You’re a woman who wanted ten fingers and ten toes, and got something more. You’re a wonder.”

A Good Good Day

Trey and Dr. Stockler, the biochemical disease doctor who oversees Trey's care. Winter 2006/2007.


We found out today. Special Access has been approved!!!!!! I am so glad it only took 2 days. I’ll start from the beginning.
This morning we saw Trey’s ENT, Dr. Moxham. He checked Trey’s tubes, which are apparently doing great. He saw clear through them (no fluid plugging them up, causing them to not work) and they are still flush against Trey’s ear drum, which means that they have not yet started to come out. Great. Dr. Moxham was also very happy with Trey’s audiology test from late December. Not only did Trey test very well (he concentrated for a long time and did everything Dreena asked, giving clear results… kids his age don’t often have a lot of understanding of or patience for testing), which is awesome in and of itself (gives me confidence with the whole brain involvement thing), but the fact that he tested well means that he doesn’t need another ABR (sedated hearing test) for the time being, and his test showed that his hearing is basically at the same point as it was for his ABR last summer (no progression of hearing loss!).
Then home for a nap. Then back to the hospital to see Dr. Stockler. First we talked about a ridge on Trey’s head which we first noticed back in August and has continued to grow since then. We had x-rays done in December, which showed that Trey has saggital craniosynostosis. Basically, one of the sutures on his head has fused prematurely. Usually, sutures stay open while the brain is growing, so that the brain doesn’t get squished. If the sutures close prematurely, it can cause pressure in the brain to build up. This is likely not the situation with Trey, as only a small portion of one of his sutures has fused, and both the surrounding sutures and his fontanel are still open. She has referred us to a neurosurgeon, however, to confirm this.
Dr. Stockler also expressed how well she thinks Trey is doing. How well his speech is developing, how coordinated he is, how well he continues to grow. She even commented that his hair is much softer than other children she has met who have MPS. As a parent who thinks about her child’s development every minute of every day, this is great to hear.
And then we talked about ERT. Now that SAP is approved, the conversation is between the Ministry and Shire to figure out how much this drug will cost over over time. I don’t know more than this, but what I do know is that both Dr. Stockler and the nurses we talked to said that ERT will begin in about 3 weeks. It still seems a bit of a ways away, but at least now it seems real.
For so long, and even after it was approved, it was this distant thing we were fighting for. Today it became real because we were given a more specific timeline and we were also given a tour of the Medical Day Unit (MDU), where Trey will get his infusions. We met the nurses on the MDU who went over everything with us. Where the food is, where the toys are, do we want a private room or a bed out in an open area…
We also went over ERT specifics. After speaking with other mamas whose kids have already started Elaprase, I have decided that I do not want Trey to go over 32mL/hour for the first few months. Protocol is that kids start off at 8mL/hr, then every 15-30 minutes are upped to 16mL, 24mL, 32mL and ending with 40mL/hr. When allergic reactions have occurred, they usually occur once the infusion rate is brought up to 40mL/hour. Not only do I not want Trey to have an allergic reaction, but once he has a reaction, they begin to pre-medicate with steroids and more antihistamines. I want to avoid that if I can. The nurse I talked with said that is okay with her.
Then we talked about how long we’ll be there for Trey’s infusion. Once a week we go to the hospital for ERT. Once there they give Trey his pre-medications (Benadryl and Tylenol). Then we wait an hour. Then, if the pharmacy is organized and on time and all that, Trey begins his infusions. Not going above a rate of 32mL/hour, we will be there for about 5 hours, and that is without hiccups like the pharmacy being on time, Trey having a reaction, the nurses having trouble accessing Trey’s Vascular Access Device (VAD…aka port) etcetera. So it will be a long day at the hospital. And one we are very much looking forward to. And if you’re still reading, you must be one of my parents or grandparents. 🙂

Still No ERT Start Date


Just frustrated waiting over here in Vancouver. Bill Mercer, the man who is helping us get Trey’s ERT from the Pharmaceutical Services Division of the Ministry of Health, is being very helpful and emailing me and talking to people when he says he’s going to. Right now I’m a little more frustrated with the medical side of things.
When we first started looking into getting a rare drug for Trey in Canada, we found out that before drugs can be brought into the country which are not federally approved (like Elaprase), Health Canada has to approve them. This process is through the Special Access Program (SAP). SAP is rarely denied because if anything goes wrong, the physician is liable, not Health Canada (It must be Trey’s physician who applies for SAP. Not the parents, not the politicians, not the government workers). SAP is separate from funding.
In order for Trey to get ERT, we knew he needed SAP and funding. All the doctors and politicians and Shire representatives and activists we talked to said SAP would not be the issue, funding would. While we were also actively trying to get the funding issue resolved, we asked our doctors repeatedly to apply for SAP, so that when funding was approved, we could move forward without having to wait for SAP to be granted. SAP usually takes 2-3 weeks to be granted. I was told, also repeatedly, that SAP was not the issue, that it could be granted in a few days, when necessary, and that we should worry about funding instead.
So guess what? SAP is holding us up because it has not been applied for. Bill Mercer told me late last week. Yeah. Frustrated. SAP was applied for early yesterday, so I have no idea how long the wait is now.
On the topic of fundraising, we have begun to organize our first official fundraisers. On Saturday, May 5, 2007 is “Tacos for Trey.” We will celebrate Cinco de Mayo with tacos and margaritas, mariachi music, pinatas, a raffle, and who knows what else. We will also be selling t-shirts for Trey (they’re in the works as we speak). Soon I will update with more info.
The other fundraiser in the works is the “Dad’s for MPS II Calendar.” It’s going to be awesome. All proceeds from all our fundraising efforts will go into the MPS II Fund within the MPS Society of Canada (all the money in the fund will go towards MPS II or related research).
I met with a charity lawyer last week, so the fund is also getting set up as we speak. If you want to donate, you can do so by cash, cheque, credit card, and soon, online donations through the MPS Society’s website (if you want to ensure that the donation goes into the MPS II Fund, make sure that it’s written on the cheque, or that you tell Kirsten that’s where you want the money to go etc.).
For the MPS Society’s address or phone number, to donate online, or for more information, please check see their website: www.mpssociety.ca. Love to you all and I hope your New Year’s are off to a good start. We’re hoping for a better 2007 than 2006!? Thank you for your continued love and support. We are so grateful and thankful for it.

"Everyone's on Holidays"

Making gingerbread houses.


This is what we keep hearing…We heard it in the summer when the FDA first approved Elaprase and we started trying to get it for Trey, and we’re hearing it now. Darned holidays!! Don’t they know they can’t go on holidays when my son needs ERT!?
I have been emailing our contacts in government every week since we made the decision to go forward with ERT. They say they appreciate and understand the urgency of the matter and “please know we are moving as quickly as we can.” For a parent waiting to begin ERT, you can’t move quickly enough.
This past week our contact in government told me he would be speaking with Shire (this past week) to make arrangements, and he would email me with the results of the conversation. When I hadn’t heard anything by Friday, I emailed him. He had not spoken with Shire. I emailed him back and asked why and apparently, “everyone is on holidays.” He said that he will be speaking with Shire next week. So, no ERT start dates yet. Looking forward to that.
I have also spoken with Dr. Stockler and she wants to wait until we get an ERT start date to organize a port placement surgery. Once we have a date, she will organize a surgery date. If the surgery is not able to happen before ERT begins, she says it’s no big deal to do the first infusion(s) through IV. For those who don’t know (and as far as I know with my very basic understanding), a port is a (hopefully) permanent device placed just under the skin that is used instead of IV during infusions. It is easier and less painful to access for Trey, than using and IV, since he will now regularly need to be pricked.

Donating Blood


Hello. We have no new news on Trey’s front, but I am writing to invite you all to donate blood or sign up to become a part of the Bone Marrow Registry program where you live.
Ryan and I have been talking and thinking about doing it for years, but we just never got around to it. Since making our decision not to go the transplant route with Trey, we have had some time to breathe and think. And we have decided to donate blood and become a part of the Bone Marrow Registry with the Canadian Blood Services: www.blood.ca.
Fortunately we are not having to face the unbelievably terrifying prospect of watching our child go through chemotherapy and transplant, and possibly not making it through that process. But for those who are going through it, and for those who know that they or their child need this treatment to live, I cannot imagine anything worse than knowing they need transplant, and not being able to get it because they cannot find a match.
It is a personal decision to give blood or donate Marrow, but I am inviting you to at least think about it. And feel so incredibly fortunate if you are not having to face a situation like this. The more potential donors we have out there, the more likely it is that children and adults will be able to find a match when they need it.
Not much news on the ERT front. Everyone’s on holidays!! I hope you are all having a relaxing and wonderful time with your family and friends during this holiday season. We wish you a Happy New Year and a whole bunch of hope and love for the New Year.

Dr. Charlie Peters


This morning I spoke with Dr. Charlie Peters, from Mercy Hospital in Kansas City. He worked for a long time with Dr. Krivit (a well known and well respected and brilliant man) in Minnesota, which is one of the biggest transplant centers in the world. Unfortunately, Dr. Krivit has passed away, but I was able to speak with Dr. Peters for over half an hour.
He reaffirmed our decision. He has not seen anything published or seen anything in his experience that shows Cord Blood is better than Bone Marrow, in any disease. As there have been only 5 Hunter children transplanted with Cord Blood and it is too early to tell whether or not these transplants worked or not, he did comment that with regards to transplant, children with Sanfilipo (MPS III) seem to have the same problem with transplant not helping the brain as children with Hunter. Many more CBT’s have been done on children with Sanfilipo and these children have continued to have neurocognitive decline.
Dr. Peters said that the medical community is not smart enough yet to know why transplant is not working for these two groups. He admitted that it is not known for sure yet that Cord Blood will not help the neurocognitive aspect of Hunter’s, but he did say that there is much evidence to suggest that Bone Marrow did not work, and there is no evidence suggesting that any of this is changing with Cord Blood.
It amazes me how willing these specialists are to speak with me. Before I was introduced into the medical world, I never fathomed I would have the courage to call or email specialists all over the world and more, I never imagined that they wold make as much time for me as they have.
Dr. Wraith emailed me back within 6 hours of my email to him and when we were at the MPS Society Conference in Ontario this past summer, he sat down with us to look at Trey’s test results to give us his thoughts. Dr. Muenzer has called me at home after me sending him an email. The same with Dr. Kurtzberg. I have been able to set up lengthy phone conversations with Dr. Szabolcs from Duke as well as Dr. Peters. Dr. Stockler returns emails on weekends. When I called Dr. Lorne Clarke to see if we could come in to speak with him about transplant, he said “Come in next week.” He has a patient wait list of months.
They are amazing. They are busy, busy people who travel he world lecturing and attending conferences, experts in their field. And though I wish we could have gone the rest of our lives without knowing what MPS is, I am glad that since this is our life, we are surrounded by such an amazing community of intelligent, helpful, compassionate, and caring doctors.

A Decision is Made


So we’ve made a decision. ERT. And there’s a whole lot of emotions which go along with that decision. It is possible that one day Ryan and I may have to make more difficult decisions in our lives than this one, but I am not sure about this. This is easily the most difficult decision I have ever made and I don’t see many decisions being more difficult. And because of this, we are exhausted. Relieved to have made a decision, but exhausted.
Since we learned about transplant as a potential option for Trey, I have really not slept a lot or done much else other than trying to have fun with the kids as often as possible. I have spoken with most of the experts in the field of transplant and MPS, I have talked to parents who have gone through with transplant and ERT, I have read their journals, I have read articles on historical transplants, we have travelled to North Carolina to see one of the leading experts in MPS II to talk about transplant and ERT, we have had transplant assessments done in North Carolina and here…
Basically, we learned that Duke is transplanting Cord Blood in children with Hunter Syndrome. We knew that BMT’s in Hunter Syndrome were stopped 10 years ago because they did not work (meaning that they did not help the brain), although after reading the research, there were not a huge number of transplants done (and the ones that were done were done on all ages and severities of disease). However, there is enough evidence for all the transplanters and MPS II specialists in the world, except Duke, to stop transplanting for Hunter Syndrome.
So we asked the question: why is Duke transplanting? Duke believes that Cord Blood is superior to Bone Marrow and they believe that this will change the outcomes of transplant in Hunter Syndrome. I want to believe this, with every part of my soul and being, and I was even willing to transplant based on a hope or belief, but… Theoretically it makes sense that Cord Blood can do more than Bone Marrow, and apparently this has been shown at a cellular level. Unfortunately, other than Duke, no one I spoke with has seen any difference in outcomes in humans or animals from Bone Marrow to Cord Blood.
This is the entire reason why we were considering transplant. Cord Blood will be different than Bone Marrow. When this idea was shot down, we began to wonder. We began talking to everyone we could. Then we heard that newer transplant procedures could make the difference. And again, most (all but two) doctors said that this will not change the outcome.
One of the doctors we spoke with, who has a way with his words…in a sick to your stomach kind of way…really hit it home for us. Basically, Duke is the only team in the world transplanting children with Hunter Syndrome. We knew this and we were okay with this, for a while. The problem is, no one else we have spoken to believes or has seen evidence that Cord Blood or newer transplant procedures will make a difference for Trey.
And although Duke is hopeful of what their transplants will show, we will not know the outcomes of the Hunter children transplanted at Duke for a few years, as they are still young and were transplanted recently. So we cannot transplant based on how they are doing. It is simply to early to tell.
And if we wait to do another assessment in February, not only are we not giving Trey the ERT that he needs right now, but if his brain is involved, we are waiting to see when his development drops as demonstrated by developmental tests, and by that time it will be too late. His brain will already be involved.
And if we did decide to transplant and it did not work, not only are we cutting at least a year out of Trey’s life to do the procedure, but we are also possibly giving Trey another disease (GvHD) on top of Hunter Syndrome, we are excluding him from any trials that may happen down the road that pertain to Hunter Syndrome, and we may also possibly kill him in the process.
We have made our decision and we are comfortable with the decision we have made, but we have very mixed feelings about it. Transplant was our only hope for Trey if his brain becomes involved. That is gone. We spent yesterday sobbing because of that.
I asked some mamas I know from the MPS forum if this ever gets any easier because although this has not been nearly as hard as adjusting to the news of diagnosis, it is definitely not easy. And it seems as though we are faced with decisions or news like this every few months.
I am so thankful for all the support we receive from the MPS families we know out there. If it wasn’t for you, I would feel very alone during these times. But on the other hand, we do not yet know if Trey’s brain is involved. And until the time that we do, I am going to believe that it’s not.
And we have been granted ERT. And we are lucky and grateful for that because we know families who are still struggling to get it. I know that life is about the lens through which you view it.
So, I will take the time I need to be sad, but I will come up from that and realize that I have amazing family and friends who have been so incredible through all of this. And I have an awesome and thoughtful and wonderful and talented and loving son who has taught me more than I could ever learn on my own and whom I love with all of my body and soul. Thank you to all of you who have helped us get this far. We love you.

My floating Head…

Christmas 2006 family photoshoot.


Well, we’re back from our appointment with Dr. Schultz and we have still made no decisions. This is not a bad thing, we actually kind of expected it. I just feel like my head is floating, spinning in circles, full of fog…
Dr. Schultz told us today that he is more uncertain about doing the transplant than he was the last time I met with him. He has spoken with all the major players in the metabolic disease transplant game as well as the major MPS doctors (Joe Muenzer, Lorne Clarke, Charlie Peters, Paul Orchard, Paul Szabolcs, Joanne Kurtzberg), and it is VERY controversial to say the least. Many centers will not even consider transplanting a child with Hunter Syndrome, and many feel very strongly about it. Apparently we have raised a few conversations around the transplant tables at meetings and conventions. ALL WE’RE TRYING TO DO IS FIGURE OUT WHAT’S BEST FOR OUR CHILD!!
So here’s what we know: Avery is a perfect match, that cute little bug. And that’s about all we know. We do know that Dr. Schultz prefers related BMT over CBT, but he will consider a CBT if we find a 6 of 6 match. He argues that because there is no hard evidence that Marrow is better than Cord Blood, and because the chance of not grafting is less with a related BM donor and because the chance of GvHD is smaller as well, he wants to do a BMT.
Also, something I did not know until today, if Dr. Schultz did do a BMT at BCCH, he would use only a partial dose of chemo (apparently Busulfan, one of the chemo’s used, is being questioned about causing Central Nervous System problems). This cannot be done with CBT, and although it slightly increases the chance of the transplant not engrafting, the lower dose of chemo is much better for Trey. Because this is the first I have heard of this, I cannot remember scientific terms or the exact pros and cons. I do know that Duke does not do the 1/2 chemo thing, they do the full blown chemo.
A positive is that Dr. Schultz thinks the mortality rate for Trey with either CBT or BMT is 5-10%. However, because Trey has CMV (a virus in his body, like the cold sore virus, that usually causes colds in a person who has a strong immune system, but can cause death in a person with no immune system), this ups the mortlity rate 1-2%.
My biggest question or concern remains over CBT or BMT. I know there is no evidence that Cord Blood is superior to Marrow, or not enough to be decisive. This is why it is controversial. What I want to know is: in 10, 20, 50 years, will there be evidence that Cord Blood is better than Marrow? If anybody out there has an educated argument for why Cord Blood is better than Bone Marrow or why they are the same, I WANT TO HEAR IT.
I understand that Dr. Schultz wants to work with evidence and he does not want to transplant based on a belief or hope or idea. However, because this is my baby, even if there is a possibility that Cord Blood is better than Bone Marrow, I want to do a CBT. Dr. Kurtzberg said: “Cord blood cells have more potential to give rise to non-blood cells in the laboratory.”
Yeah… So what’s next? In February, we go to Duke for a follow-up developmental assessment with Dr. Escolar. Dr. Schultz is very interested to see how Trey does in that.
Also in February, Dr. Schultz is attending a meeting which will be reviewing the article that Dr. Kurtzberg and the Duke team of doctors are publishing about the five children with Hunter Syndrome who have had Cord Blood Transplants. Although Dr. Schultz has read the abstract for this article, he does not believe it is convincing enough for him to do a transplant for Trey. It speaks very briefly about a stabilization or improvement in neurocognitive abilities. Dr. Schultz wants to read the article and hear the presentation before making any definite decision. This is fine with us because we need to get Trey’s developmental test with Dr. Escolar done before we make any decisions anyways.
So now it’s all happening in February. And then we’ll meet with Dr. Schultz on February 16 to make final decisions (for real this time…). So now I just have to read all the articles ever published on transplant in people with Hunter Syndrome, and hopefully get in touch with Dr. Peters, who has much historical information on this. And talk with Duke about all this…and make appointments with Duke for the assessment.
At least now we know what our next steps are. A bit of a relief, although it will be an even bigger relief to make the final decision (although I am now much more comfortable with waiting…playing with Trey is so much fun!!).
In other news, Trey is starting to speak like crazy. It’s really awesome and great and wonderful. And he’s also become way less aggressive than he was a couple of months ago (maybe it was a phase?). These recent happenings allow me to entertain the possibility that maybe Trey does not need a transplant after all.

Conversation with Dr. Kurtzberg

Summer 2004. Trey and Papa (my dad).


I spoke with Doctors Kurtzberg and Szabolcs this morning from Duke. Where to start…if Avery is not a 6 of 6 match, she thinks that there will not/should not (I’m not sure which) even be a conversation about BMT or CBT, so she suggested waiting until we know how much of a match Avery is before worrying. Although, she said that even if Avery were a perfect match, she would still do a CBT. She still holds that cord blood is better than bone marrow, regardless of newer transplant procedures. She believes that because it is younger, it can do more. She said that looking at children with Hurler’s (MPS I), she thinks that the kids transplanted with cord blood have higher IQ’s, better bones and joints, and do better overall than kids transplanted with bone marrow. There’s a lot of other stuff she said, but I want to wait to find out more before I post. She will also be seeing Dr. Schultz this weekend at a Conference, so she said she’d talk with him about Trey. Another conversation I had today was with MSP. MSP has agreed to pay for the medical expenses from our trip to North Carolina! They will not cover the travel or accommodation, but at least some of it will be paid for.

CONTACT US AND SHARE YOUR STORY!

We want to hear from you! Send us your story so we can share it with the world. Every person and family battling Hunter Syndrome has a unique story to tell - one of bravery, resilience, and perseverance. We'll share all stories online here and on our social media feeds!