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A hopefull trial   Message List  
Reply | Forward Message #136 of 592 |
Hi everybody.I found this article.It is hopefull isn't it:

Treatment with Intravenous Immunoglobulin Improves Muscle Strength in
a Patient with Dysferlin-Deficient Muscular Dystrophy

Simone Spuler, Joanna Zabojszcza, Male Friedrich-Freksa, Berlin,
Germany

OBJECTIVE: To investigate whether ivig improves muscle strength in
dysferlin-deficient muscular dystrophy. BACKGROUND: Recently, we
reported that complement inhibitory factor CD55 is downregulated in
skeletal muscle of mice and patients with dysferlin-deficient
muscular dystrophy (LGMD2B). The absence of CD55 increased
susceptibility of dysferlin-deficient human myotubes to complement
attack in vitro (Wenzel et al. J Immunol. 2005 Nov 1;175:6219-25).
One of the possible therapeutic effects of intravenous
immunoglobulines (ivig) is inhibition of complement factors C4 and
C5. We therefore tested whether ivig might be beneficial in LGMD2B.
DESIGN/METHODS: As compassionate use a single female 32 year old
patient with dysferlin-deficient muscular dystrophy was treated with
ivig (Intraglobin, Biotest AG, Taunus, Germany) for six months after
informed consent was obtained. After an induction cycle of 0.4 g/kg
body weight for 5 days ivig was applied once a month at 0.4 g/kg body
weight. Muscle strength was tested by manual muscle testing. A muscle
biopsy specimen was analysed before and after treatment for
deposition of sarcolemmal C5b9 membrane attack complex. RESULTS: Ivig
was tolerated well. After three months the patient reported a marked
improvement in her ability to walk and in her strength when holding a
heavy bag. Muscle strength improved further until treatment-month
six. All muscle groups tested improved during treatment, some muscles
such as the tibial anterior muscle improved from grade of paresis of
2/5 to almost full strength at 4-5/5. C5b9 membrane attack complex,
present on almost all muscle fibres in the pretreatment muscle
specimen, were nearly absent in muscle obtained after treatment.
CONCLUSIONS/RELEVANCE: The effect of ivig in LGMD2B should be tested
in a controlled clinical trial.








Wed May 10, 2006 11:35 am

izzetbesi
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Message #136 of 592 |
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Hi everybody.I found this article.It is hopefull isn't it: Treatment with Intravenous Immunoglobulin Improves Muscle Strength in a Patient with...
izzetbesi
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May 20, 2006
12:08 am

That's good news... I am wondering what happend to the rest of the group... Nobody posts lately. Maybe everybody got cured and they run away... Was a lady in...
AIR-COM
vlacas2002
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May 20, 2006
2:20 am

I posted a couple of times but the moderator didn't look at my posts and so they did not show. AIR-COM <matanas@...> wrote: That's good news... I...
John B
bluedestin
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May 20, 2006
4:55 pm

Thank you very much!!! This gives me hope! I want a cure so bad! izzetbesi <izzetbesi@...> wrote: Hi everybody.I found this article.It is hopefull...
John B
bluedestin
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May 20, 2006
4:55 pm

Yes, I thoroughly agree that it is very hopeful, thanks for sharing it with the members. I had come across this abstract a few weeks ago. Since then, I have ...
baw1064
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May 21, 2006
4:39 am

Hi. I agree that anything new is exciting. I was previously diagnosed with Polymyositis & had a week long course of Intravenous Immunoglobulin as part of...
Suzanne Croft
craftycroftyuk
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May 21, 2006
11:54 am

I've been lurking on this list--too busy to post anything lately! Although granted, it's hard to define "lurking" on list with such few posts. Brad Williams...
Sharon Hesterlee
shesterlee
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May 22, 2006
4:46 pm

Four years ago, when I was diagnosed with LGMD 2B, Dr. Karpati from Montreal prescribed IVIG. I had two back to back treatments, but then I moved and my new...
Amanda
inquisitivem...
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May 24, 2006
11:53 pm

Amanda, That's very interesting, and thanks for telling about your experience with IVIg. Since right now we're trying to figure out if some type of trial is ...
baw1064
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May 25, 2006
1:37 am

Hi Brad, I can't remember if Dr. Karpati treated other dysferlinopathy patients with IVIg but I wouldn't be surprised if he had. He is among the world's...
Amanda
inquisitivem...
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May 25, 2006
3:03 am

Amanda thank you for sharing your experience.It is very important and im agree.I think it was ivig.I want to ask some quesions.Can you reply at here or to my...
izzetbesi
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May 27, 2006
5:14 pm

That is correct, at the time my condition was not 100% confirmed. My biopsy showed a possible depletion of the dysferlin signal. As my file says: "In view of...
Amanda
inquisitivem...
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May 27, 2006
10:09 pm

So nice to hear from you Amanda. I wana get some of that too... I found this info in the following link. ...
AIR-COM
vlacas2002
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May 25, 2006
2:02 am
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