Gosh, I don’t blog a lot these days. There’s a lot of reasons for that: 1. Trey is doing well, so my need to write with updates or for cathartic reasons is not as often (of course, this is relative and speaking from rare disease world, as last week Trey had ERT Tuesday, sedated tooth extractions Wednesday, Intrathecal ERT under general anesthetic Thursday and left yesterday with Ryan to UNC for clinical trial testing). 2. I’m not doing as many fundraisers. 3. I have been and am making a conscious effort to live presently and watch and be instead of spending so much tim up in my head (which leads to writing ;-)). With that said, have I got some updates for you!!
First, our family’s ONLY fundraiser of the the year, the Scotiabank 5K & 1/2 Marathon is on Sunday, June 26!! Please join us to run or cheer or donate! If you want to run or cheer, send me a message for more info. To sponsor a runner, you can check out “Team Trey’s” page and donate to any of the runners! Click here to check out who’s running for Team Trey.
If you’re on the fence about donating, here’s a couple things to consider: 1. MPS is rare so we don’t get as much love (or donations or awareness) as other more common diseases 2. Rare disease research is completely privately funded- there is not government support- so every dollar matters!! Because of this, I am SO grateful to the Higginson’s extended clan (http://www.jacksjourney.ca/) and JF Aublet & Edith Lacroix’s family (Hope for Felix-Antoine, Felix-Antoine is pictured above) for joining The Isaac Foundation and fundraising for the MPS II Fund. It takes a lot of the pressure off and brings so much community and sense of family not only to our fund, but the entire Isaac Foundation. 3. The Isaac Foundation puts your donations straight to research. Check out The Isaac Foundation’s CRA status here, it’s something we are very proud of!
If you haven’t seen our Grants page yet this year, it’s worthwhile to take a look. On January 31, 2016, with the awesome support and generosity of The Isaac Foundation, our MPS II Fund granted $62,000 to Dr. Douglas McCarty at Nationwide Children’s Hospital for his MPS II gene therapy program (we started funding his research back in 2013). There are so many reasons I’m really excited about Dr. McCarty’s work:
- Dr. McCarty’s wife, Dr. Haiyan Fu, has led the way with gene therapy research in MPS III, which means that they have worked out many of the kinks with applying to the FDA, with pharmaceutical companies etcetera, so that the MPS II program is moving speedily and steadily along.
- Dr. McCarty gives us regular updates and is very down-to-earth and transparent with his process, which builds trust, hope and excitement!
- Dr. McCarty has gained the support of MANY MPS II organizations all over North America, which enables his research to go faster and farther!
- And last but not least, Dr. McCarty’s research is promising and is getting darned close to human clinical trials.
One example: Emily wrote up a science lesson on self-driving cars. Trey features in her writing as “a boy I (she) knows, that when he grows up, a self driving car will give him independence he couldn’t have.” I was in tears. It’s a simple reflection, but I know from listening to her talk about him, there is a changed world view behind those words.
The advantage of Trey in MT Deb is completely ours to share.”
And if you’re not completely bored yet, below are a few links I have shared on Facebook, in case you’re not on Facebook or missed them.
An article from the Wall Street Journal that reminds me I am a dragon mom. Trey may be doing well, but that is because of a drug in clinical trial for a rare disease. This article strikes that all too familiar fear into my bones and reminds me that I will likely always need the ability to breathe fire for my boy: http://www.wsj.com/articles/mental-dystrophy-at-the-fda-1461885588
An update on Isaac McFadyen, the child behind The Isaac Foundation, our friend, and a boy who is only weeks younger than Trey and was diagnosed just months before him: https://www.theisaacfoundation.com/stop-this-train-an-update-on-isaac/
A song written by MPS II mom, Melissa Hogan, which helped raise a welcome amount of awareness for MPS II and was nominated for numerous awards (grab a tissue for this one too): https://www.youtube.com/watch?v=F7MubmW7dxU
Love to you on this first day of May. XOXO Deb