today perusing the MPS forum and reading the journeys of other families struggling with this disease. I stopped reading a few minutes ago and I must say that I am very hopeful for Gracie and her chances at this point. I can't believe how lucky we are to have gotten our diagnosis so early and that we have the opportunity to begin treatment so quickly.
Gracie goes for her second ERT infusion bright and early on Wednesday morning... her first one here in Florida at Shands. The doctor overseeing her treatments is Dr. Susan Staba-Kelly (nice last name!) who I understand previously did transplants for Hurler patients at Duke University. I am excited to meet her and get her take on Gracie's treatment plan.
I am also excited to continue the ERT treaments and watch Gracie's progression. Other MPS parents have noted that they have seen physical changes in their children on ERT, most notably the specific physical characteristics associated with the disease. It makes me wonder if Gracie's looks will change much. I hope not too much because I think my kid is cute as a button as she is, but it will be interesting to see what will happen.
Subscribe to:
Post Comments (Atom)
9 comments:
Can somebody tell the MPS society about this hope-inspiring forum?
Great googly-woogly, that is some depressing organizational web site! I have read some pretty sad stories about this disease, and I know that, in many instances, hope is in short supply. Does anyone out there have an affiliation with this organization?
Would they possibly be interested in undertaking an information overhaul?
I know that most of the members of the forum are on the MPS Society registry and also on the Director's Board. I can't imagine that they don't know.
I will say that there are lots of sad stories there as well, but I have chosen to focus on the brighter side right now.
The MPS Society board of directors are all parents of children with MPS. I agree that the mps society site is a grim site to visit, and is not at all a good resource for newly diagnosed families.
My guess is that they don't have time or resources to update. :( There is some pretty good information out there, it just takes a lot of googling and searching to track it down.
When Lauren was diagnosed, I was handed a print-out of information from the MPS Society and sent home with it with almost no explanation. I couldn't believe what I was reading. My doctor had printed out a death sentence for my daughter from the Internet and told me to take it home and read it. The site is very bleak.
I think that part of the problem we have with some of the 'fact-based' sites as parents of children with MPSI is that we have hope. We can be hope-inspired. MPSI has a life-saving treatment even though it does not have a cure.
Also, many of these treatments that we have now are so new and so experimental (until fairly recently) that the prettier side of the picture is harder for them to write about... they want to give facts.
Lastly, and this is totally my opinion, but I came to the conclusion early on that much of what I read about MPS and the lack of treatments and such... was aimed at being a place where one could donate money to research. In which case, a brighter side of the picture doesn't serve them well. And I don't mean that to disparage those who put the site up at all... I'm just saying that they have to let people know what the worst case scenario is to bring in funding.
As for the ERT, I think with Gracie being so young, you won't notice as many changes as she doesn't have as much build up of GAGs in her body yet. Maybe across the bridge of her nose and in her ears... where cartilage will start to soften. But I think either way Gracie will stay cute as a button.
Oops, I think my comments might be longer than your blog. :-) ... Have a good night.
Interesting remarks. I guess my thought on this would be that, since Noodles and GM have already done some legwork (and Kindall, too) is there a way that some already-compiled updates could be presented in a format that MPS could easily access and distribute?
It seems to me that the "facts" have evolved somewhat, and I would hope that donors would still want to fund research that was actually having an impact where the rubber meets the road.
There would also be plenty of opportunities for allocating funds for innovations in patient/family services for these extended courses of treatment and the subsequent lifetime of related medical interventions that appear to be the norm.
I feel certain that the MPS society must be interested in advancing their cause, and in supporting families. I must admit that I haven't done the kind of homework Kindall and GM have done, but if there was interest, I would certainly collaborate on a project to make some of this information available at MPS society, in what is perhaps usually the first place a newly-diagnosed family is sent.
This seems like an opportunity for a really important contribution to patient and family welfare that is just waiting to be taken on.
I agree with both of you. I'm sure funding in allocation are huge issues and barriers to maintaining the information.
I also agree that we could help TD, although I hesitated before offering up my own time to do the legwork, tee-hee.
One thing we could definitely do is provide digestible summaries of current lit, good contact info for treatment centers that focus on MPS and links to the clinical trial site. Those could be quick hits and really useful for newly diagnosed families.
Another issue is that the board probably are fairly far away from being newly diagnosed, so their information needs and top of mind issues may have less to do with immediate treatment needs and more to do with living with the disease. Just a thought. Let me know how I can help. I'd love to leave a mark that might help others.
I have nothing to add on the MPS society bleakness, treatment info or any of the other highly intelligent stuff you guys are discussing. I just wanna say thanks for a blog update and kiss that baby for me!
I'm sorry I haven't posted yet, but I want to thank you for sharing Gracie's journey. I am so glad you, Jimmy and Gracie are blessed with such a wonderful support in your family Sarah. I love you and Gracie so much. Give her a big kiss from me!!!
KelleyKR
I agree with test driver here, I think that it is a disservice to those parents for are looking for that one that thing- a shred of hope, after being handed such a grim picture. Not to mention direction, options, and a deeper understanding.
Mack has done a bang up job with his line by line explanations, in laymans terms, that anyone can understand.
I for the life of me can't understand why the website is so darn dismal.
I have a chronic disease, and our national web page I find can be overly rosy on the "reality" of the disease, but truth told, for the newly diagnosed, it once again provides what they need most, and that is some notion of hope.
Enough said.
Post a Comment