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Tami
March 9th, 2004, 04:02 PM
Edie's Mom writes.......................

I'm wondering if any of you know of kids on the treatment who are no longer experiencing some of the benefits. Edie is 5, started ERT in May, and had a huge increase in energy initially. Now she (again) has almost stopped walking. I'd love to hear from someone with children who were pretty severely affected before starting treatment

Tonia Karl
May 14th, 2004, 10:37 PM
Taylor has not had any changes since starting ERT in Dec. She is not severly affected but her hands are still getting worse. Genzyme is now going to do a GAG test on her to monitor the buildup as I am getting frustrated. Our next test is a Pulmonary Function Test and boy do I hate new test. They always seem to bring more unexpected bad news. When she had her ECHO they found out she had a Bicuspid and also a Murmor which they didn't know about before and of course she had thickening of the heart walls. I hope we all see more results soon. It is so frustrating!!!!! :8):

3coolkidsmom
May 18th, 2004, 11:56 PM
Tonia,
I am wondering if Taylor has had hand surgery yet for carpal tunnel syndrome. All three of my children had the surgery at age six and it made a big difference in their fine motor skills.

Also, with our daughter Maddie who started ERT at age 10, it took probably six months or more before she said she felt better. ERT seemed to help our other two children a little more quickly.

3coolkidsmom
May 19th, 2004, 12:15 AM
Tonia,
P.S. Sorry, I just thought about this. I think you may be disappointed if you are searching for "changes" in Taylor because that is not really how Aldurazyme works, at least not for the majority of patients who have been using this drug. Aldurazyme slows the progression of many of the symptoms of the disease and helps to stabilize others. Some of my friends who are parents of Aldurazyme patients describe therapy as "stopping the disease in its tracks", or"preventing further damage". Of course, we know that it does not treat the orthopedic issues or the CNS disease. And, we also know that Aldurazyme can not go back and correct all of those things in the patient's body that occurred before the use of the drug, like carpal tunnel. Taylor may need to see a neurologist for an EMG study to determine the need for surgical intervention. It is better to do this sooner rather than later or Taylor may suffer permanent loss of function in some area of the hand or fingers. Get it checked out if you haven't done so already.
Sorry I'm beeing so bossy and pushy.I am going right now into the kitchen to take some midol. Maybe two or three of them! Please forgive me. I'll write you back tomorrow when my mood has been altered!

3coolkidsmom
May 19th, 2004, 12:48 AM
Tami,
Because Edie has Hurler syndrome, she has some orthopedic issues that differ from Hurler-Scheie and Scheie patients. Hurler patients must deal with spinal deformities such as kyphosis and scoliosis, hip dysplasia & genu valgum (knock knees). Even with ERT, these things continue to worsen as well as Edie's CNS disease. She may not feel like walking because she is having ortho progression or neuro changes. Her parents need to make sure she is being dosed correctly and they need to speek to their ERT coordinator about these issues.

ediesmom
June 25th, 2004, 03:05 PM
This is Laura, Edie's mom. I don't know how Tami's picture ended up next to my name...maybe because I'm too computer challenged to figure out how to load my own picture.
Anyway, Edie's regression continues and I can only assume it due to CNS deterioration. Dr. Muenzer suggested ruling out spinal cord compression but we need to put her under sedation to determine if that could be the problem...and then we problably would choose not to treat it anyway. Since she is happy we are not doing anything invasive.
I just hoped I might get some validation of my own assumptions. Thanks for your thoughts Amy and others.

Jessica
June 25th, 2004, 05:07 PM
Laura,
Tami was just bringing the topic over from the old forum that you had asked back in March so it appears like you wrote the message but Tami actually copied and pasted it from the old hang out!

I encourage you to make a post in the Introduction forum and let all the members on the forum know more about you and your family! I believe I saw your photo in the new Courage issue! I was Ooohing and Awwwing over Edie’s photo. What a doll baby!

I hope you can visit more often. I would love to get to know you better and would especially like to know more about Edie!

(I feel another Miss Smiley coming on :D )

Jessica
Hurler Mom to Kaity

silverwolf
November 5th, 2004, 03:27 AM
They ami
I have been on the enzymereplacemnt for mps2 for aboutt hree years and before the treatment i had baasically no stamina and i was in a wheel chair wherever i wnet. i could walk a little but like through a small store at the mall then i would go back to the wheele chair and get pushed to the enxt store. I would still need a nap and painkiller afterwords though After the ezyme i got a lot of my stamina back and my joints are loser. Although i haven;t expierence any regressions for the improvemnts. although other areas have been regressed. my eyesight got really bad in the matter of months and that was turing I was getting the treatment. I'm also haven't isues with the joint in my right knee. Not sure if this was the kind of info you where looking for but i thought i'd mention it

mike

ediesmom
November 8th, 2004, 12:34 AM
Hi all,
I feel inclined to update you on Edie. She is continuing to experience regression, but nothing significant like we witnessed this past winter. Edie is such a happy camper even though she now struggles to hold up her big ol' noggin. A few months ago we started increasing her weekly dosage of Aldurazyme. Currently she is getting 5 vials instead of the traditional 3. If we ever get her initial reports from Dr. Muenzer then we will compare GAG levels from her first few months of treatment to those six months ago to more current levels. I'll keep the Forum informed if we have any revelations.

Rita
December 21st, 2004, 12:45 PM
Hi Laura,

I am Rita, Benjamins mom. He is 10 has Hurler and started ERT on 16th April. He has responded well to the treatment and his GAGs was redused from apprx 330 to 15,3. Did you get any feed back on Edies GAGs status? How is Edie doing now?

Love
Rita :kiss:

Barbara Pryor
December 22nd, 2004, 06:10 PM
Laura,
I saw Edie at the conference and just wanted to say that she is so cute. I just love her hair and that smile that she has. :)

ediesmom
March 6th, 2005, 11:00 PM
As I mentioned several months ago we increased Edie's dosage of Aldurazyme. First we added one extra vial (making the dose 4 vials) and after about six weeks we added one more vial. We stayed at 5 vials for several months and over the course of this time we periodically had urinary and blood samples drawn.
Now after much waiting (on Genzyme) we finally have results which indicate that neither her urinary GAG or her antibodies seem to be affected by the increase in dosage. Her GAG levels increased initially and then went down and then went up slightly. :?
So this week she'll be back at 3 vials and only 4 hours instead of 6!
I hope this information may be useful to anyone considering a change in dosage.
-Laura

Tressia
March 8th, 2005, 06:21 PM
My son has Hunter Syndrome, however, he has been on ERT for over 3 years now.

Before the ERT he used a medical stroller regularly and tired easily. Since the ERT he has more energy and walked anywhere he wanted to go. But, withing the last 6-8 months his walking has declining to the point that he now crawls when he is at home.

When we entered the clinical trial they did tell us up front that the ERT would not benefit the blood/brain issues nor bone issues. So I am assuming what we are dealing with is the bone issue. My son still has his energy, more range of motion in his arms, hands, and more than he had in his legs. His heart has stabilized and not changed since being on ERT. So in our case the benefits of ERT are still worth it.

GOD Bless,

Tressia