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Six months on Cystadane

Asking for prayer. Ethan has been on Cystadane (betaine) for the last six months. This is the medicine that Dr. Braverman (PBD specialist from McGill University in Canada) is working on getting a clinical trial started with soon. Ethan has been on the medicine for six months and tomorrow Ethan has the blood draw that will "determine" if it is "working." It will take a few weeks to get the test results back but the hope of course is that when they come back that there will be an improvement in his VLCFA and plasmagen levels. We pray that the Cystadane is not just working for Ethan but also for the future possiblilities for all children with PBD. This has been a huge leap of faith for us as Ethan is the only child in the country with PBD taking Cystadane at this time. The medicine is an FDA approved medicine for a different unrelated metabolic disorder, but still, it is a little scary having Ethan be the first.

We'll keep everyone posted! Thank you for your your thoughts and prayers.
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  1. AnonymousJuly 20, 2012 at 7:44 AM

    Hi,
    My name is Marcelo and I am from Brazil.
    A friend of mine’s son (six months old) got a particular disease, methylmalonic aciduria with homocistinuria.
    His doctor has prescribed Betaine and my friend has ordered it at a compounding pharmacy as it is the only way to get it here in Brazil.
    It tastes very badly, so he is having problems to have his baby taking it.
    We were looking in the internet to see if there was another choice abroad and found Cystadane!
    As you are giving your baby this medicine for some time, we would like to know if it tastes not that bad and how is Ethan taking it! Did you have the same problem at the beginning?
    We wish your family all the best.
    Thanks for sharing your story with us; we wish your family all the best!

    Marcelo
    magonnu@hotmail.com

    ReplyDelete

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