If you would like to donate and have questions, send me a message.
Deb & the Purcell’s
If you would like to donate and have questions, send me a message.
Deb & the Purcell’s
When Joe Geluch, President of Naikoon Contracting (the company that built our house), approached me last summer after getting to know our family through the build of our house, to put on a fundraiser for our MPS II Research Fund, I thought it was a kind gesture, but never did I have any idea that it would be brought to fruition. Fruition was Sunday, April 22, 2018.
$24,000 was raised for our Fund. For research. For our boys with MPS II. I don’t give as many updates on Trey as I did when he was first diagnosed, and although Trey is mostly stable due to the IV & IT treatments he receives, our life is still far from typical. It’s hard to describe, to a large group of people, what our life is like unless you see us regularly. What do you focus on? What and how much do you say? Trey’s progression due to the build up of GAGs as a result of MPS II is minimal to none after he began receiving Intrathecal ERT over seven years ago, but he still has MPS.
This past year, two issues have arisen for Trey that at times have gotten me pretty down:
One, we found out Trey has Obstructive Sleep Apnea (OSA). There’s no surgical treatment, no cure and since November 2017, we’ve been working with his Behaviour Analyst to get him to wear a C-Pap machine at night. Due to his OSA, Trey is tired most days and now due to our routine trying to get him to wear a C-Pap machine at night, misses most of his night time activities like floor hockey, bowling and softball, which he loves. If he doesn’t learn to tolerate the C-Pap machine, that could lead to heart issues due to lack of oxygen when he obstructs.
The second, which has a longer history than a year but we thought was resolved, is a severe limp with no known cause, other than MPS (he has hip dysplasia as a result of MPS). It was awful to the point of Trey not being able to walk for weeks at a time last summer, but somehow resolved on its own. Then a week ago, it came back with full-strength after the one activity he still participates in because it’s early enough to get him home for his C-Pap routine, soccer. He could barely walk (I contemplated a wheelchair because I don’t think he could use crutches) from Monday night until Thursday morning and then was fine. Sunday night after Construction Cares, the limp was back. Avery and Sadie helped carry him back to the couch when he’d try to get up. It is so hard to see. We now might have to take him out of soccer. His life is already limited by MPS cognitively, with multiple trips to hospitals here and in North Carolina, and with weekly infusions, it gets me down to see his quality of life (which I think is pretty good, he might just be the happiest member of our family) further impacted by MPS.
I say all this with the hopes of you understanding just how much it means to our family that you showed up yesterday, in whatever form that was. MPS can be sad and hard and lonely and it is you who lifts us up when we are down. Even to those of you who couldn’t make it but sent a text, that matters. You remembering matters. You asking how we’re doing despite us being twelve years into this journey, matters. To all who came and donated and volunteered and sponsored and attended as guests, you honestly fill our hearts. You make it so we can pick ourselves up and say “We’ve got this. We can do it. We are surrounded.” Ryan and I left Construction Cares last night knowing we’d never be able to fully express how thankful we are. We didn’t get to thank each of you and even if we did, it wouldn’t be enough. I hope my words can in some small way help you know what it means to us that you came.
Thank you to everyone from Naikoon who volunteered. It was SO cool and overwhelming to walk into The Pipe Shop and see all these people we know, all these people who began conversations with us years ago about building a sustainable and durable house for our family, to the people who actually built our house and know the guts of our house better than we do, to the people who emailed and organized and showed up for the build of our house when we had questions, and to the people from Naikoon we didn’t know until this fundraising began! We are so touched by how involved you all were. It took a team. Thank you.
And an extra special thanks goes out to Madeleine Funk, Tyler Waddell, Pamela Buck and Joe Geluch. Gosh, when I think about how stressed and anxious and insanely driven I was putting on other events, and then I think of what you all did for me, for us yesterday, I am forever grateful. Joe, we will never EVER forget what you have done for us.
We will share more pictures from the event in the coming weeks, and here is a link to a CBC video, our clip starts at 10:34: http://www.cbc.ca/player/play/1216906307541.
This indoor fair is for adults and children of all ages, with games and activities hosted by local businesses. Bike Valet and admission are by donation, and activity tickets will be on sale for $2 each.
Activities include: a Construction Station sponsored by Naikoon Contracting Ltd.; Decorating Station sponsored by Pro Works Painting; Gardening Station sponsored by LifeSpace Gardens & GardenWorks; and Face Painting sponsored by KYBE Electrical Contracting.
There are super-fun games too, all with a prize for the highest score of the day, including a Spider Boxer game sponsored by Griffins Boxing; Electronic High Striker Game & Kiddie High Striker Game side by side (so adults and children can play together); and an Xtreme Skateboard game.
Also look for our bike valet sponsored by The North Shore News; music from Tall Music DJ Services; hourly 50-50 draws from our emcee Kevin Evans; and some amazing prize baskets put together by local businesses that are being raffled for $2 a ticket.
Everyone welcome, rain or shine, with net proceeds from this event benefitting Hunter Syndrome/MPSII research.
People have pointed out to me in the past day that gene therapy is coming up if this trial does get shut down. One: this trial is not getting shut down unless it’s over my jailed or dead body. Two: Gene therapy is hopeful and likely, but I will not rest my child’s life on hope and possibility. Nobody knows this better than families who have kids with MPS II Trey’s age and older. When Trey was diagnosed at the age of two in 2006, IT trial research was already going on. I spoke with Dr. Muenzer about it every few months. Every spring we were told human clinical trials would begin in the fall. Every fall we were told it would begin in the winter. Every winter we were told it would begin in the spring. For three years. During that time, most of Trey’s peers fell outside of the IT trial criteria and therefore did not qualify.
So yes, gene therapy will hopefully come to fruition, but I will not let a drug that is holding my child stable and saving his life, go, with the hope for gene therapy. I don’t know gene therapy will come, and if it does come, I don’t know if Trey will qualify for the trial, and I don’t know if it will come in time to save Trey.
Trey has IT therapy. I am not giving that up until or unless there is another treatment that is available and works.
I’m not going to say a lot right now as I’m still processing this news and I’m not sure how much I can/should/want to be posting on the big wide web. What I do know without a doubt is that this drug works and I will go to jail before I see this drug get taken away. Melissa Hogan has issued a Clinical Trial Alert that contains more information, please see below.
If you have questions, don’t hesitate to reach out.
There are opportunities for sponsorships, donations for gift baskets, and volunteering, so if you own or are part of a company or would like to be involved in a different way, please let me know.
Up until now, the vast majority of fundraisers have been in my hands, which holds with it a lot of stress and pressure. I remain blown away by the generosity of Joe and everyone who has joined us this early on in the event. I feel relief and that this is not all on me and gratitude that we can continue to raise necessary funds for research despite me not doing the lion’s share of the work.
Best of all for all of you, with the awesome ideas of our core committee, this event is going to be incredible, with fantastic food, treats and activities. Check out the attached poster, mark your calendars, and continue to check in with the Facebook page to get exciting updates!
Thank you for your support, year after year.
Deb, Ryan, Trey and the rest of our Purcell crew!
Along with a few other MPS II parents, I spoke with our gene therapy researchers, Dr. Doug McCarty and Dr. Haiyan Fu, a couple weeks ago. Mice with MPS II start dying around 7-months of age. When Dr’s Fu & McCarty treated their mice that made it to 9-months old with double the dose of what they give their younger mice, these 9-month old mice came back. They normalized. I don’t know how many months old my Trey mouse would be, but can you imagine? I don’t even know what that would look like (to be clear, I don’t want Trey as anybody other than who he is, but avoiding early death, surgeries, hospital trips, and letting him be as independent as he can and wants to be would be my dream). Trey as a typical 13-YO kid? I won’t. I can’t. Not yet. It’s too scary. The letdown would be too monumental. Even writing this I’m choking back tears and emotions. Shove them back down. For now.
BUT, I do believe it is possible. WITH YOUR HELP. The thing is, with a rare disease, we NEED your help raising awareness. Nobody knows about MPS II Hunter Syndrome but us and in order to raise $2.5 Million dollars, we need people to know.
With gene therapy SO close, our MPS II community has banded together. We’ve organized teams. If each team raises $10,000, we’ll reach $1 Million. I think we can do better. Our runs raise $10,000 each year without a cure on the horizon. Tacos for Trey raised $20-40,000. Our gala raised $80,000. $10,000 is attainable. Let’s beat $10,000. $10,000 to see a Trey who has no physical or cognitive limitations?? I’d give a f@*k of a lot more than that.
Our MPS II community has also created a documentary series about MPS II Hunter Syndrome to raise awareness. The first of the episodes will be released on May 15- International MPS Awareness Day. Here’s the trailer.
So what can you do? 1. Watch the trailer. 2. Along with the trailer, share a link to our Scotiabank Run fundraising page:
Ask your community to donate and tell them why they should donate. Tell them how close we are to human clinical trials. Tell them how you know Trey and how MPS II Hunter Syndrome affects him. Tell them how MPS II Hunter Syndrome effects you.
THANK YOU. If you have questions, ask. I’m a mom on a mission to save her kid’s life. I’ll do anything. The fire-breathing dragon has been mostly sleeping for the past few years, but she’s being called back out… it is time.
There are so many “Day’s.” Anti-bullying Awareness Day, World Thinking Day, Earth Day, World Cancer Day… google it and you’ll find days you didn’t even know existed! Rare Disease Day is one that matters to me, probably obviously, but you may not know why.
People don’t donate to causes they don’t know about. Why would they? Your grandma had Alzheimers, your mom has breast cancer, your uncle had a heart attack, your friend has diabetes. If someone is going to donate to a cause, it’s one they’ve seen hurt the people they love. The more people who have something that needs curing, the more people who know about it and the more people who will donate to it. Which is a good thing- except when it comes to rare diseases, and which is why we need YOUR help.
Aside from a few companies who manufacture rare disease drugs or doctors who recall MPS being vaguely mentioned during the one day in med school that covered lysosomal storage diseases, the vast majority people who know about MPS are the ones who’ve run into kiddos with it. YOU. And the only way we can raise awareness about rare diseases so that people who don’t know Trey can say: “Yes, I know a rare disease called MPS! My friends’, neighbours’ brother has it,” is by talking about them. A LOT. Tell people how MPS affects YOU. Is Trey your neighbour? Your student? A patient? A member of your community? A friend? What do you see when you see Trey and our family? Why does he need a cure? YOU need to talk about it and it needs to be personal. Especially if they’ve never met Trey, you need to give them a reason to care.
So this is why Rare Disease Day matters to me. It’s a day I can ask that you share something about MPS on Facebook, at work, with a friend, at a class you’re teaching… Show them pictures of Trey or Trey’s website. Talk about the impact MPS has had on your life.
Please. Do it today or do it any day between now and February 28. Or maybe every day. Mark it in your reminders. Talk about MPS. So we can raise awareness about this and other rare diseases. So we can find a freaking cure for this soul-crushing disease that takes our boys away from the families who love them- from the people who would die for them if it meant their child would live. Rare Disease Day Matters.
For more information on Rare Disease Day, go to: http://www.
PS. You may notice the poster was made by the Mesothelioma Cancer Alliance. Rare diseases unite!!
This picture is of Trey 4-months before diagnosis, at 19-month of age.
Trey was diagnosed 11-years ago today. I was scared of moms like me when Trey was first diagnosed- moms who knew the insides and outsides of MPS. Hearing moms talk about seizures and g-tubes and cognitive delay made me want to throw up and hide. I didn’t want this life. I wanted normal. I wanted ignorance. But now that I’m in it, it’s more full of love and openness and friendship than I ever could have imagined. Scary and sad it is, but that’s life. The “before” me would have learned that eventually. So here it is. D-Day. A day that changed my life. And although meaningful and significant, it is no longer a day that feels like the end of the world. It’s a day where I got to drive Trey to a school he loves. A day that I get to pick him of from a school he loves. A day that I get to talk to him and hug him and tonight, give him kisses as high as he can count- through giggles. A day like that in rare disease world, I would take forever.