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Has anyone had any experiece with this? My infant has Beta-KetoThiolase Disorder and I asked her Doc about this and he said it may be an option. I am looking to see if anyone out there has ever done this?
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Bone Marrow Transplant for Organic Acidemia?
- By Kristinna
- Posted February 3, 2009 at 6:12 pm · 3 replies
- In News and research
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- By oaanews
- Posted February 4, 2009 at 12:01 pm
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Hi Kristinna -- I have not heard of a bone marrow transplant treatment for any organic acidemia disorders. Does your doctor have a medical journal article where this has been done before? It's my understanding that all organic acidemias are autosomal recessive (genetic) and therefore there aren't any 'cure' except maybe gene therapy.
Might want to ask your question on the OAA Listserv?
Kathy Stagni
mom to Melissa, Propionic Acidemia
- By Kristinna
- Posted February 4, 2009 at 1:17 pm
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I found some websites that discussed BMT's with acidemias. It has been done sucessfully with propionic but has never been tried with BKT since is is more rare.. Check out the following website for one reference:
http://www.marrow.org/PATIENT/Undrstnd_Disease_Treat/Lrn_about_Disease/Meta bolic_Storage/index.html
- By oaanews
- Posted February 5, 2009 at 7:39 am
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Hi Kristinna - thanks for sending the link - the article in the link is referring to 'storage' metabolic disorders - which is different 'set' of metabolic disorders....and I didn't see propionic listed as having been treated with BKT - send me what you've seen on PA being treated with that - since I haven't seen it. I have not heard of BKT being a treatment option for OA's....thanks!
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